The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• Formation Bio launched a $600m fund to accelerate drug development using AI.

• Anthropic’s IPO filing revealed its own drug-discovery unit and biology lab.

• Amgen’s Imdelltra cut lung cancer death risk by 58% as first-line maintenance.

• Lilly’s retatrutide beat Ozempic on blood sugar and weight loss in diabetes.

• The FDA drafted a single-licence path for bespoke gene editing treatments.

Browse by day:

• Weekend catch-up: Roche · Amgen · AZ · BMS CAR-T · Hengrui · Moderna

• Monday: Pfizer · Anthropic · BioNTech · Keytruda · Abbott

• Tuesday: FDA gene editing · Novartis · Artiva · Ferring-Gameto · Enhertu

• Wednesday: Formation Bio · retatrutide · BGI-MGI

• Thursday: Lilly · Dren Bio

• Friday: Bambusa-Aspen

Weekend catch-up

Bristol Myers Squibb disclosed a patient death in its phase 1 trial of the CAR-T therapy zola-cel in multiple sclerosis.

The patient developed neurological symptoms after infusion and died of septic pneumonia 44 days later, the day after receiving high-dose cyclophosphamide. No other deaths have been reported among 190 patients treated in zola-cel’s autoimmune trials. BMS said the event was unrelated to its earlier decision to pause enrolment over a separate safety signal, which it says it has now identified. It is working with regulators to restart trials.

• Breakfree-2 results for zola-cel in MS at MSToronto · Bristol Myers Squibb

• BMS sheds light on patient death in CAR-T trial · Fierce Biotech

Tempus AI signed $192.5m in data agreements with Bristol Myers Squibb and Pathos AI, alongside a $237m financing for Pathos led by BMS.

BMS will pay Tempus $137.5m for wider access to oncology data, while Pathos will pay up to $55m for records of 500,000 patients with Alzheimer’s disease and other neurological conditions. Pathos will use the data with its AI drug-development platform, which BMS has also licensed. The agreements run for approximately five years. Pathos’s financing valued the company at about $3bn.

• Tempus AI Form 8-K on BMS and Pathos agreements · SEC EDGAR

• Pathos AI secures $237m Series E financing · Pathos AI

Hengrui’s trastuzumab rezetecan more than doubled progression-free survival against chemotherapy in HER2-low breast cancer.

In the 551-patient phase 3 HORIZON-Breast06 trial in China, published in JAMA and presented at ESMO, median progression-free survival was 16.4 months against 6.8 (HR 0.27), with benefit in both hormone receptor-positive and -negative disease. Severe treatment-related side effects, mostly low blood counts, hit 84.1% of patients against 67.9% on chemotherapy, and half needed a dose cut.

• Trastuzumab rezetecan in ERBB2-low breast cancer: HORIZON-Breast06 · JAMA

Further phase 3 data on Moderna and Merck’s melanoma vaccine showed overall survival remains immature.

In INTerpath-001, intismeran autogene plus Keytruda reduced recurrence or death by 42% and distant metastasis or death by 38%. The overall survival hazard ratio was 0.77 (p=0.33), not yet statistically significant, and follow-up continues. The vaccine was manufactured for more than 99% of patients assigned to receive it, with no deaths attributed to the vaccine.

• Recapping the phase 3 INTerpath-001 results · Moderna

• INTerpath-001 data detail survival, subgroup and safety findings · CancerNetwork

Amgen’s Imdelltra cut the risk of death by 58% as first-line maintenance in small cell lung cancer.

In the phase 3 DeLLphi-305 trial in extensive-stage disease, presented at ESMO on Saturday and published in NEJM, adding Imdelltra (tarlatamab) to AstraZeneca’s Imfinzi (durvalumab) extended median overall survival to 21.1 months from 12.7 on Imfinzi alone. Nearly 80% of patients on the combination were alive at one year, against 53%. Amgen is sharing the results with regulators worldwide.

• Imdelltra with Imfinzi reduced risk of death by 58% in first-line ES-SCLC · Amgen

• Tarlatamab–Durvalumab Maintenance for Extensive-Stage SC Lung Cancer · NEJM

Roche’s divarasib cut the risk of death by 44% against a pooled control of the two approved KRAS G12C drugs.

In the phase 3 Krascendo 1 trial in previously treated KRAS G12C-mutated non-small cell lung cancer, presented at ESMO on Sunday and published in NEJM, median overall survival was 17.7 months on divarasib against 12.0 in the control arm, where 156 patients took Amgen’s Lumakras (sotorasib) and 15 took Bristol Myers Squibb’s Krazati (adagrasib). Median progression-free survival was 9.8 months against 5.7. Fewer patients stopped divarasib because of side effects: 4.8% against 11.7%.

• Roche’s divarasib showed best-in-class efficacy in KRAS G12C lung cancer · Roche

• Divarasib vs sotorasib or adagrasib in KRAS G12C–mutated NSCLC · NEJM

AstraZeneca reported significant survival benefits in two phase 3 lung and gastric cancer trials at ESMO.

In SAFFRON, Tagrisso plus Hutchmed’s Orpathys (savolitinib) cut the risk of death by 32% against chemotherapy in EGFR-mutated non-small cell lung cancer that had become MET-driven on Tagrisso (median 17.8 against 12.9 months), and the risk of progression or death by 45%. Hutchmed said a US application has been filed.

In CLARITY-Gastric01, the antibody-drug conjugate sonesitatug vedotin cut the risk of death by 53% in third-line and later Claudin 18.2-positive gastric cancer (10.0 versus 5.0 months). The reduction was 42% across all patients treated in second line or later, although the other primary endpoint, progression-free survival, missed significance.

• Tagrisso plus Orpathys cut death risk by 32% in MET-driven NSCLC · AstraZeneca

• HUTCHMED highlights SAFFRON Phase III data at ESMO 2026 · HUTCHMED

• Sonesitatug vedotin cut the risk of death by 42% in CLARITY-Gastric01 · AstraZeneca

Monday 26 October

BioNTech/DualityBio’s HER2 ADC gave only a modest benefit over chemotherapy in a phase 3 trial in hormone receptor-positive, HER2-low breast cancer.

DYNASTY-Breast02 met its progression-free survival endpoint, but interstitial lung disease was more common than in earlier trials, with some severe and fatal cases. The partners said the benefit to patients is limited and that they will decide next steps.

• DYNASTY-Breast02 global phase 3 topline results · DualityBio

Abbott launched a whole-genome version of its Oncodetect molecular residual disease (MRD) test for solid tumours.

The tumour-informed blood test tracks up to 5,000 patient-specific variants, against 200 previously, and uses MAESTRO technology to achieve a 30-fold improvement in its analytical detection limit. In colorectal cancer data presented at ESMO, sensitivity for recurrence within two years improved to 72.2% from 42.2%, using a single post-surgical blood sample. Oncodetect came with Abbott’s $21bn acquisition of Exact Sciences in March, giving it an established position in the rapidly growing cancer diagnostics market.

• Abbott launches whole-genome sequencing Oncodetect MRD test · Abbott

• Abbott presents new ESMO data on Oncodetect sensitivity · Abbott

• Abbott completes acquisition of Exact Sciences · Abbott

The FDA approved Pfizer’s Talzenna plus Xtandi for HRR gene-mutated metastatic hormone-sensitive prostate cancer.

In the phase 3 TALAPRO-3 trial, the combination cut the risk of radiographic progression or death by 52% against Xtandi plus placebo. Overall survival data remain immature, and severe anaemia occurred in 51% versus 3% of patients. The combination was approved in castration-resistant disease in 2023.

• Talzenna plus Xtandi FDA-approved for earlier metastatic prostate cancer · Pfizer

• FDA approves talazoparib + enzalutamide for HRR-mutated prostate cancer · FDA

Keytruda alone beat carboplatin-paclitaxel chemotherapy as first-line treatment for mismatch repair-deficient endometrial cancer in a phase 3 trial.

In KEYNOTE-C93, presented at ESMO and published in The Lancet, median progression-free survival was 38.2 months on Keytruda against 8.4 on chemotherapy. Overall survival favoured Keytruda numerically but not significantly, with 85.2% of patients progressing on chemotherapy subsequently receiving immunotherapy. Severe treatment-related adverse events were less frequent (18% versus 51%). The trial did not compare Keytruda alone with the current standard of chemotherapy plus immunotherapy.

• Keytruda KEYNOTE-C93 phase 3 results in endometrial cancer · MSD

• First-line pembrolizumab vs chemo in dMMR endometrial cancer · The Lancet

Anthropic’s IPO filing revealed an expanding drug-discovery operation, including its own molecular biology lab.

In the S-1 it filed with the SEC on Monday, Anthropic says it has acquired Coefficient Bio, hired industry experts, built a molecular biology lab and developed AI agents to support drug development. In a separate update on Tuesday, Anthropic reported early results from six collaborations with pharmaceutical and biotechnology partners, including experimental antibody engineering and an 89% reduction in manufacturing-document review time at Enveda. It is also pursuing its own drug-discovery programmes for rare childhood muscular dystrophies. Most work remains preclinical.

• Anthropic, PBC Form S-1 registration statement · SEC EDGAR

• Early progress on accelerating drug development with Claude · Anthropic

Tuesday 27 October

Novartis took a $1.9bn impairment on del-desiran after it missed its primary endpoint in phase 3.

Del-desiran came with Novartis’s $12bn acquisition of Avidity in February. The drug missed its primary endpoint of video hand opening time in the phase 3 HARBOR trial in myotonic dystrophy type 1, despite activity on secondary measures. The impairment helped cut reported Q3 operating income 61% to $1.8bn, while core operating income rose 10%, sales grew 4% to $14.5bn, and full-year guidance was raised. CEO Vas Narasimhan said Novartis may launch new drugs in Europe’s private or self-pay markets because of US most-favoured-nation pricing, Endpoints reported.

• Novartis delivered sales growth and core margin expansion in Q3 · Novartis

• Novartis considers bypassing European payers due to MFN · Endpoints News

The European Commission approved Enhertu after surgery for HER2-positive early breast cancer with residual disease.

It is Enhertu’s first EU approval in early breast cancer. In the phase 3 DESTINY-Breast05 trial, Enhertu cut the risk of invasive recurrence or death by 53% against Kadcyla. At three years, invasive disease-free survival was 92.4% versus 83.7%.

• Enhertu approved in the EU for HER2+ early breast cancer · AstraZeneca

Artiva abandoned its NK cell therapy after two trial deaths and disappointing response durability, and will cut 94% of its staff.

Artiva stopped developing AlloNK, an allogeneic natural killer cell therapy given with Rituxan in rheumatoid arthritis and other autoimmune diseases. One trial participant died of leukoencephalopathy assessed as likely fludarabine toxicity, and a second death was judged unrelated. Longer-term durability also fell short of Artiva’s threshold. The company reported cash equivalents of ~$319m at 30 September, and is weighing liquidation options. Its board approved the wind-down on 22 October.

• Artiva discontinues development of AlloNK · SEC EDGAR

• Artiva Biotherapeutics Form 8-K · SEC EDGAR

Ferring Pharmaceuticals agreed to buy fertility start-up Gameto in a deal worth up to $1.4bn.

Gameto’s Fertilo uses stem cell-derived ovarian support cells to mature eggs outside the body, potentially shortening IVF cycles and reducing the need for hormone injections. It is available in Australia and parts of Latin America and is in phase 3 in the US. The acquisition value depends on development, regulatory and commercial milestones, with no upfront payment disclosed. The deal has yet to close.

• Ferring and Gameto sign agreement for proposed acquisition · Gameto

• Ferring to acquire next-gen IVF startup Gameto · BioPharma Dive

The FDA issued draft guidance on how individualised gene-editing products can be approved.

Sponsors could submit multiple patient-specific variants in one biologics licence application (BLA) and add new variants after approval, potentially without further clinical trials. The proposed framework could make treatments for ultra-rare genetic diseases more commercially viable by allowing regulatory evidence to be shared across variants of a therapy.

• Individualized human genome editing products: BLA considerations · FDA

Wednesday 28 October

Eli Lilly’s retatrutide beat Novo Nordisk’s Ozempic (semaglutide) on blood sugar and weight loss in a phase 3 trial in type 2 diabetes.

In the 1,298-patient TRANSCEND-T2D-2 trial, retatrutide reduced HbA1c by 2.2 percentage points against 1.8 on Ozempic 2 mg at 80 weeks. Weight loss was 19.5–20.9% versus 9.4%. Discontinuations due to side effects were higher at 4.8–7.9% versus 2.7%. Retatrutide remains unapproved, and detailed results have yet to be published.

• Lilly’s retatrutide outperforms Ozempic in head-to-head phase 3 trial · Eli Lilly

Formation Bio launched a $600m fund to license stalled drug programmes and develop them with AI.

The Formation Bio Drug Development Accelerator Fund will license phase 1 and 2 drug programmes into separate companies, each supported by Formation’s clinical development teams and AI systems. Partners may receive upfront payments and retain a share of future milestones and royalties. Chief executive Ben Liu told Forbes the fund aims to advance 10 to 15 programmes over three to five years.

Investors include research foundations, endowments and hospitals. The structure gives Formation access to development capital without diluting shareholders in the parent company. Formation has separately raised $615m at a reported $1.8bn valuation. Whether its AI-enabled development model can improve clinical productivity at scale remains to be demonstrated.

• $600 million debut fund to expand drug development capacity · Formation Bio

• Why an AI biotech launched a $600 million fund to buy stalled drugs · Forbes

The US Department of Homeland Security added BGI Group, BGI Genomics and MGI Tech to its Uyghur Forced Labor Prevention Act entity list.

DHS alleges that the Chinese genomics companies’ genetic-testing activities supported forced-labour programmes in Xinjiang. Goods produced by listed entities are presumed to involve forced labour and are barred from US import unless that presumption is rebutted. BGI Genomics denies the allegations. The restrictions could further constrain MGI’s ability to compete with Illumina in the US sequencing market.

• Notice of the updated UFLPA Entity List · Federal Register

• BGI Genomics statement on its inclusion · BGI Genomics

Thursday 29 October

Dren Bio raised $350m to fund late-stage development, regulatory submission and a planned launch of its lead antibody dibotatug.

Juvia Group and Lightspeed Venture Partners co-led the round. Dibotatug targets CD94 to selectively deplete pathogenic cytotoxic cells and is being developed for cytotoxic T-cell lymphoma and large granular lymphocytic leukaemia, two rare blood cancers with no specifically approved therapies. Dren previously received $600m upfront from Sanofi for a separate B-cell depletion programme.

• Dren Bio announces $350m financing to advance dibotatug · Dren Bio

Eli Lilly’s Q3 revenue rose 39% to $24.4bn, and it raised 2026 guidance to $91bn-$92bn.

Mounjaro (tirzepatide) revenue rose 65% to $10.7bn, while US sales of Zepbound, its obesity brand, rose 48% to $5.3bn. Together, the two brands accounted for approximately two-thirds of Lilly’s revenue. The new guidance range lifts the midpoint by $5.5bn (6%), although quarterly revenue benefited from approximately $900m in milestone payments and favourable rebate adjustments.

• Lilly reports third-quarter 2026 financial results · Lilly

Friday 30 October

Bambusa Therapeutics and Aspen Neuroscience priced Nasdaq IPOs expected to raise $168.9m and $140.6m.

Bambusa, which develops long-acting bispecific antibodies for inflammatory diseases, upsized its offering to 10.6m shares at $16, the midpoint of its range. Aspen, which is developing induced pluripotent stem cell-derived therapies for Parkinson’s disease, priced 9.4m shares at $15, the bottom of its range. Both are scheduled to begin trading on 30 October, with the offerings expected to close on 2 November.

• Bambusa Therapeutics announces pricing of upsized IPO · Bambusa Therapeutics

• Aspen Neuroscience announces pricing of IPO · Aspen Neuroscience

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• Moderna and Merck’s vaccine cut recurrence or death risk by 42%.

• EQT agreed a €10.8bn take-private of Qiagen.

• GSK’s hepatitis B drug Hibsago received FDA approval.

• US Treasury proposed curbs on outbound biotech investment.

• Merck KGaA agreed to buy Silence Therapeutics for $1.3bn.

Browse by day:

• Monday: Silence · Roche-Hansoh · Disc · Jemperli · EyePoint · City

• Tuesday: Hibsago · Myriad-Labcorp · ICER · Sanofi-ScionxBio

• Wednesday: Qiagen · Cencora · Abbott · Thermo · Ultomiris · CE marks · AI Proteins

• Thursday: Moderna · US Treasury · Roche · Gilead-PepStar · AbbVie

• Friday: Lycia · J&J-Smith & Nephew

Monday 19 October

Merck KGaA agreed to buy London’s Silence Therapeutics for $1.3bn.

The German group will pay $20 in cash per ADS, a 130% premium to Friday’s close, valuing the equity at $1.3bn and the enterprise at $1.1bn. It gains divesiran, an siRNA drug for the blood disorder polycythaemia vera that is heading into phase 3. The UK scheme of arrangement is expected to close in Q1 2027.

• Agreement to be acquired by Merck KGaA · Silence Therapeutics

Roche licensed ADCs from China’s Hansoh for $400m upfront and >$15bn in contingent milestones.

Hansoh also receives $110m on the deal’s first anniversary, plus tiered royalties. The collaboration covers two preclinical ADCs and additional programmes against Roche-proposed targets. Hansoh will take them through phase 1a before handing development outside mainland China, Hong Kong, Macau and Taiwan to Roche. Hansoh retains rights in those territories. The deal is the partners’ second in ADCs.

• Collaboration with Roche to advance novel ADC portfolio · Hansoh

Disc Medicine’s bitopertin met every primary and secondary endpoint in a phase 3 trial in erythropoietic protoporphyria.

In the 183-patient APOLLO trial, Disc reported a 41.4% placebo-adjusted reduction in blood protoporphyrin IX. Patients gained 12.2 additional pain-free hours in light during month six vs placebo. Disc plans to resubmit its FDA application by end 2026.

• Success on all endpoints in phase 3 APOLLO · Disc Medicine

The FDA granted accelerated approval to GSK’s Jemperli (dostarlimab) for MSI-high or mismatch repair-deficient locally advanced rectal cancer.

It is the first immunotherapy approved for these stage II and III patients and could spare some of them chemotherapy, radiation and surgery. In the phase 2 AZUR-1 trial, 68% of evaluable patients (98/144) achieved a sustained clinical complete response at 12 months without surgery. No disease recurrences were reported among responders after a median follow-up of >2 years.

• Jemperli approved for MSI-H/dMMR locally advanced rectal cancer · GSK

EyePoint’s vorolanib eye insert missed the primary endpoint in its second phase 3 trial in wet age-related macular degeneration.

Preliminary topline results from LUCIA failed to establish that the six-monthly insert was non-inferior to Eylea (aflibercept) given every eight weeks. Patients gained 2.5 letters of vision against 6.1 with Eylea, while receiving 38% fewer injections after loading doses. EyePoint is now focused on its diabetic macular oedema trials, which are expected to read out in Q4 2027.

• LUCIA phase 3 trial: DURAVYU in wet AMD topline results · EyePoint

City Therapeutics closed its Nasdaq IPO, raising $230m.

The RNAi developer sold 12.78m shares at $18, including the full exercise of the underwriters’ option. Trading began on 16 October under the symbol CTY.

• Closing of $230m upsized IPO, including underwriters’ option · City

Tuesday 20 October

The FDA approved GSK’s Hibsago, a six-month hepatitis B treatment aimed at functional cure.

Hibsago (bepirovirsen) is a first-in-class antisense drug. In the B-WELL phase 3 trials, 19% of patients in the approved population reached functional cure: HBV DNA and HBsAg remained undetectable for at least 24 weeks after stopping all treatment. Standard antivirals alone typically achieve <1% functional cure after a year. The drug received priority review and Breakthrough Therapy designation. Patients can inject themselves after the first dose.

• Hibsago (bepirovirsen) approved in the US for chronic hepatitis B · GSK

Myriad Genetics agreed to sell its prenatal testing business to Labcorp for $210m in cash.

Labcorp expects the business to add about $160m of revenue in year one. The deal should close by early 2027. The proceeds exceed half Myriad’s pre-announcement market value, and it expects the disposal to improve its growth and profitability.

• Agreement to sell prenatal health testing business to Labcorp · Myriad

The median net launch price of new US drugs rose 35% in real terms from 2022 to 2025, to $257,000 a year, ICER and West Health found.

About one in four drugs launched in 2025 cost >$500,000 a year net. Only about one in three newly approved drugs previously reviewed by the Institute for Clinical and Economic Review were priced at or below its value benchmark. During the first year after approval, only a third of outpatient prescriptions were filled within 30 days of an attempt to fill them.

• Median prices of newly launched drugs continue to exceed $250,000 · ICER

Sanofi partnered with Cambridge, UK, start-up ScionxBio to make longer-lasting versions of some of its mRNA candidates.

ScionxBio, formed through a split of Eleven Therapeutics, will optimise the molecules with its TERA platform through in vivo validation. Sanofi will then take over development and commercialisation. The disease areas are undisclosed.

• Collaboration to develop long-acting xRNA therapeutics · ScionxBio

Wednesday 21 October

EQT agreed to take Qiagen private at a €10.8bn enterprise value.

The Swedish buyout firm’s EQT XI fund offers €46.50 a share in cash, a 40.3% premium to Qiagen’s price before takeover reports surfaced in July. The enterprise value is equivalent to about $12.1bn, including net debt. ADIA and CPP Investments are co-investing. Qiagen’s boards recommend the offer. It needs acceptances from >50% of shares and should close in mid-2027, subject to regulatory and other conditions.

• Agreement on recommended all-cash takeover offer with EQT · Qiagen

Cencora agreed to buy American Oncology Network, a physician-led group of >350 cancer care providers, at an enterprise value of up to $2.4bn.

Cencora already owns about 4%. If existing stockholders retain a quarter of the equity, it expects to pay about $1.7bn to increase its ownership to 75%. The enterprise value includes up to $100m in contingent value rights.

• Acquire American Oncology Network to strengthen community oncology · Cencora

Abbott plans to spin off its nutrition business, which has $8.5bn in annual sales and brands including Ensure and Similac.

The planned tax-free distribution to Abbott shareholders should complete by the end of 2027. Daniel Salvadori, who runs established pharmaceuticals and nutrition, will lead the new company.

• Intention to separate global Nutrition into an independent company · Abbott

Thermo Fisher Scientific’s Q3 revenue rose 9% to $12.1bn (+6% organic).

Adjusted EPS rose 15% to $6.68.

• Thermo Fisher Reports Third Quarter 2026 Results · Thermo Fisher Scientific

Roivant’s Lisraya (brepocitinib) met its primary and all key secondary endpoints in two phase 3 trials in non-infectious uveitis.

Across the 371 patients in CLARITY-1 and CLARITY-2, median time to treatment failure exceeded 48 weeks on the drug, against about 12 weeks on placebo in each trial. In the pooled analysis, 62% had inactive disease without steroids at week 48, against 20%. Priovant plans a US filing in H1 2027.

• Positive CLARITY-1 and CLARITY-2 results in non-infectious uveitis · Roivant

The FDA granted accelerated approval to AstraZeneca’s Ultomiris to reduce proteinuria in adults with primary IgA nephropathy at risk of progression.

Approval rests on a 43.4% placebo-adjusted reduction in urine protein at 34 weeks, from an interim analysis of the phase 3 I CAN trial. Whether the drug slows kidney function decline is not yet established.

• Ultomiris receives US accelerated approval in IgA nephropathy · AstraZeneca

The UK government confirmed it will keep accepting CE marked medical devices in Great Britain indefinitely.

CE marked (EU approved) devices make up >90% of those registered for use in Great Britain and will not require a separate UKCA approval. The UKCA mark remains available as the domestic route. The consultation drew >1,000 responses.

• Government confirms indefinite recognition of CE marked medical devices · MHRA

Astellas signed a miniprotein drug discovery deal with AI Proteins, which is eligible for >$400m in upfront and milestone payments.

Astellas holds an exclusive worldwide licence to resulting candidates and will handle development and commercialisation. AI Proteins would earn royalties on any sales.

• Collaboration with Astellas to develop miniprotein therapeutics · AI Proteins

Eli Lilly’s triple agonist retatrutide reduced obstructive sleep apnoea severity in two phase 3 trials published in NEJM.

In adults with overweight or obesity but without diabetes, the weekly 9mg dose reduced the apnoea-hypopnoea index by a mean 34.3 events an hour at 80 weeks, against 9.9 on placebo, from a baseline of 58.6. In patients with type 2 diabetes, the corresponding reductions were 24.7 and 10.3 events an hour.

• Retatrutide once weekly for treatment of obstructive sleep apnea · NEJM

Thursday 22 October

Moderna and Merck’s personalised cancer vaccine cut melanoma recurrence or death risk by 42% in phase 3.

In INTerpath-001, intismeran autogene, an mRNA therapy tailored to each patient’s tumour, was added to Keytruda after surgery for stage IIB–IV melanoma. At a median 24.6 months’ follow-up, estimated two-year recurrence-free survival was 82.2%, versus 70.0% with Keytruda alone (hazard ratio 0.58). The risk of distant metastasis or death fell 38%. Overall survival data are not yet mature. The results have been accepted for publication in NEJM. The companies are discussing the data with regulators.

The data leaked online on Thursday, prompting the release two days before the scheduled ESMO presentation. Moderna’s shares fell about 25% intraday. Moderna had risen over fourfold following August’s initial data. Analysts at UBS and William Blair characterised the pullback as a sell-the-news reaction following that rally.

• Results from phase 3 INTerpath-001 at ESMO 2026 · Moderna / Merck

• Shares fall on eagerly anticipated cancer vaccine results · BioPharma Dive

The US Treasury proposed adding biotechnology to its curbs on outbound investment.

The draft rules implement the Comprehensive Outbound Investment Security (COINS) Act, passed in the 2026 National Defense Authorization Act. US investors would have to notify Treasury of certain transactions involving companies linked to countries of concern and would be barred from others. The Act names five technology sectors. The Treasury Secretary added biotechnology to the proposed regulations. Treasury is seeking feedback on the draft.

• Proposed rule to implement the outbound investment security act · US Treasury

Roche’s sales rose 7% CER in Q3, and it confirmed its 2026 outlook.

Nine-month group sales grew 6% CER. Diagnostics sales rose 4% to CHF10.3bn, as core lab and pathology demand outweighed China’s healthcare pricing reforms. Roche now aims to bring up to 20 new medicines to patients by 2030. The day before, Roche announced pooled phase 3 data showing its oral MS drug fenebrutinib reduced annual relapse by 55% against teriflunomide in relapsing MS.

• Third-quarter sales growth of 7% at constant exchange rates · Roche

• New neuroimmunology trial data at MSToronto2026 · Roche

Gilead agreed to pay $1.275bn upfront for PepStar Therapeutics’ oral IL-23 receptor inhibitor programme.

Lead drug HCXT-2001 is a clinical-stage oral macrocyclic peptide. Gilead sees potential as a daily treatment for IBD, alone or with emvistegrast.

• Acquire oral IL-23R inhibitor HCXT-2001 to strengthen IBD pipeline · Gilead

AbbVie’s lutikizumab met its phase 3 endpoint in hidradenitis suppurativa.

At week 16, 31.4% of patients reached HiSCR75, against 23.4% on placebo. This requires at least a 75% reduction in the combined abscess and inflammatory nodule count, with no increase in abscesses or draining fistulae. The 1,400-patient INTREPID trial also met eight ranked secondary endpoints.

• Phase 3 INTREPID topline results for lutikizumab in HS · AbbVie

Friday 23 October

Lycia Therapeutics priced its enlarged Nasdaq IPO to raise $318m.

The company priced 17.64m shares at $18, with trading expected to begin on Friday under the symbol LYCA. The offering is expected to close on 26 October. Lycia develops drugs that degrade disease-causing proteins outside cells. Its lead IgE degrader is in phase 1 for allergic disease.

• Pricing of upsized initial public offering · Lycia Therapeutics

• Lycia Therapeutics amended Form S-1 registration statement · SEC EDGAR

Talks to merge Johnson & Johnson’s orthopaedics business with Smith & Nephew collapsed over who would run the combined company, the FT reported.

• How a transatlantic mega-merger fell apart · Financial Times

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• Stryker agreed to invest $400m in CMR Surgical, with an option to buy the rest.

• Bristol Myers Squibb reported two positive phase 3 readouts in a day.

• The EMA backed RevMed’s Rasonque in metastatic pancreatic cancer.

• OMass Therapeutics raised $310m to take an adrenal drug through phase 2.

• The FDA set out to rebuild how it regulates rare disease drugs.

Browse by day:

• Weekend catch-up: Natera · UK VPAG · Asahi Kasei · AZ

• Monday: BMS · Stryker-CMR · Steris

• Tuesday: Roche-Rona · BioNTech · Liquidia-United Therapeutics · reverse mergers

• Wednesday: FDA · Vir-Gilead · Verastem · Galderma-ZO · GSK · Alzheimer’s · Iambic

• Thursday: OMass · OrbiMed · AZ-insitro · Rasonque EU · Labcorp · Roche Enspryng

Weekend catch-up

Natera’s multi-cancer early detection blood test received FDA Breakthrough Device designation.

The assay analyses circulating cell-free DNA to screen for multiple cancers, extending Natera’s oncology ambitions beyond its established Signatera molecular residual disease (MRD) monitoring business. The FDA considered data across 12 cancer types, including sensitivity, specificity and the ability to identify a cancer’s tissue of origin. Natera did not disclose performance figures. The designation provides an expedited development and review pathway, but does not constitute regulatory approval.

• FDA grants Breakthrough Device designation for MCED assay · Natera

Principles agreed for the UK’s next branded medicines pricing scheme include a seven-year term, newly published minutes show.

The Department of Health and Social Care (DHSC) released minutes of a July review of the voluntary scheme for branded medicines pricing, access and growth (VPAG) with the Association of the British Pharmaceutical Industry (ABPI). Principles agreed for the successor scheme cover growth, innovation, uptake, competition and investment links. Government will work with companies on strategically important medicines, including flexible commercial and risk-sharing models.

• 2024 VPAG operational review: minutes from 23 July 2026 meeting · DHSC

• UK’s next pricing and access scheme to last for 7 years · pharmaphorum

The FDA approved Asahi Kasei’s Hovilpri (pritelivir) for mucocutaneous herpes simplex lesions refractory to standard antivirals in immunocompromised adults.

The oral tablet is approved where lesions are refractory, with or without documented resistance, to acyclovir, valacyclovir or famciclovir. Asahi Kasei says it is the first US herpes simplex treatment with a new mechanism in nearly 30 years and plans to launch it by the end of 2026.

• FDA approval of Hovilpri for refractory mucocutaneous HSV lesions · Asahi Kasei

AstraZeneca’s efzimfotase alfa missed statistical significance in a phase 3 trial in hypophosphatasia.

In HICKORY, presented on Sunday at the American Society for Bone and Mineral Research meeting, patients aged 12 and over who had never taken Strensiq (asfotase alfa) walked 17.05 metres further than on placebo in six minutes at week 25 (p=0.1675). AstraZeneca said the lack of statistical significance was largely due to better-than-expected results in the adult-onset placebo group.

• Efzimfotase alfa numerical 6MWT improvement in HICKORY Phase III · AstraZeneca

Monday 12 October

BMS reported two positive phase 3 readouts, one in idiopathic pulmonary fibrosis and one for an alpha-emitting radiopharmaceutical.

RYZ101, acquired through the $4.1bn 2024 purchase of RayzeBio, improved progression-free survival against standard treatment at the ACTION-1 interim analysis. Patients had gastroenteropancreatic neuroendocrine tumours that progressed after lutetium-177 therapy. BMS says these are the first phase 3 results for an alpha-emitting radiopharmaceutical in any indication and will discuss a filing with the FDA.

In ALOFT-IPF, the 60 mg twice-daily dose of admilparant, an oral LPA1 antagonist, slowed the loss of lung capacity against placebo at week 52. The 120 mg dose also slowed the decline but missed significance. BMS plans regulatory submissions.

• Positive topline interim Phase 3 ACTION-1 results for RYZ101 in GEP-NETs · BMS

• Phase 3 ALOFT-IPF trial of admilparant met the primary endpoint · BMS

Stryker agreed to invest ~$400m in CMR Surgical, with an option to buy the whole company.

The deal marks Stryker’s entry into soft-tissue surgical robotics, challenging Intuitive Surgical’s dominant da Vinci platform. Stryker will take preferred equity in the Cambridge, UK, company and become exclusive US sales agent for its Versius robots, extending its existing Mako orthopaedic robotics business. CMR’s modular Versius system has been used to treat more than 45,000 patients worldwide, although its US clearance is currently limited to gallbladder surgery. CMR retains development and sales outside the US. The transaction remains subject to regulatory approval.

• Strategic alliance with CMR Surgical to advance robotic-assisted surgery · Stryker

• Versius passes 45k patients treated globally ahead of US expansion · CMR (March)

Steris agreed to buy Xodus Medical, a Pennsylvania maker of specialty surgical products, for $2.1bn in cash.

Xodus makes operating-room consumables, including its Pink Pad patient-positioning systems. Steris expects 2026 revenue of $245m and adjusted EBIT of $135m. The deal includes an estimated $300m of tax benefits, reducing the effective purchase price to $1.8bn, with up to $150m more payable if Xodus meets performance targets in 2028. Steris will fund the acquisition with debt and expects to close by end 2026.

• Expands healthcare consumables with agreement to acquire Xodus Medical · Steris

Tuesday 13 October

Rona Therapeutics licensed its ALK7-targeting RNAi obesity drug to Roche for up to $100m in upfront and near-term milestone payments.

Rona can earn up to $1.06bn more in milestones, plus royalties. ALK7 sits mainly in fat tissue, and Rona says blocking it could cut visceral fat while preserving lean mass. The drug uses Rona’s platform for delivering RNAi beyond the liver.

• Global license with Roche ALK7-targeting RNAi in obesity · Rona Therapeutics

BioNTech’s mRNA cancer immunotherapy BNT113 did not significantly improve progression-free survival in a phase 2/3 head and neck cancer trial.

AHEAD-MERIT tested BNT113 with Keytruda (pembrolizumab) as first-line treatment for unresectable recurrent or metastatic, PD-L1-positive, HPV16-positive head and neck cancer. On its monitoring committee’s advice the trial continues to overall survival, its second primary endpoint. A gain there would still make the trial positive. The setback follows BioNTech’s decision in August to discontinue a phase 2 trial of its personalised mRNA cancer vaccine BNT122 in colorectal cancer, contrasting with Moderna and Merck’s positive phase 3 melanoma results that month.

• Phase 2/3 BNT113 update in HPV16-positive head and neck cancer · BioNTech

Liquidia and United Therapeutics settled all six lawsuits between them, with Liquidia paying a net $381m and giving up Yutrepia’s PH-ILD indication.

Liquidia can continue selling inhaled treprostinil Yutrepia for pulmonary arterial hypertension. It has submitted an FDA supplement to remove pulmonary hypertension associated with interstitial lung disease from the label and will no longer promote the drug for PH-ILD.

• Global settlement resolves United litigation and preserves Yutrepia in PAH · Liquidia

Biotech start-ups are turning to reverse mergers to fast-track their stock market listings, the Financial Times reported.

The FT counted 23 reverse-merger announcements in 2026, up almost sixfold from four in 2025, matching the number of traditional biotech IPOs. The transactions exploit a pool of listed biotechs left with depleted pipelines following clinical failures, often retaining cash and a stock market listing. Specialist life sciences investors are increasingly backing these mergers with substantial new financing, turning what was once considered a backdoor listing into an alternative route to the public markets.

• Biotech start-ups turn to reverse mergers to fast-track listings · Financial Times

Wednesday 14 October

The FDA launched an initiative to rebuild its regulation of rare disease drugs.

The Rare Disease Framework Initiative, set out in a white paper, aims for rules fitted to each disease, its patients and the evidence a small population can realistically produce. Only about 5% of rare conditions have an FDA-approved treatment, the agency said. It promises clearer, more consistent decisions and risk-based manufacturing reviews. A public meeting on 28 October opens consultation, which could lead to new guidance, revised regulations and organisational changes.

• Rare Disease Initiative to develop fit-for-purpose drug development framework · FDA

Vir Biotechnology agreed a collaboration with Gilead to discover three dual-masked T-cell engager candidates for solid tumours.

Vir gets $90m upfront, made up of $65m in cash and a $25m equity investment at a 30% premium. Up to $45m in near-term milestones and $1.74bn later can follow. Vir designs the candidates. Gilead develops and sells them.

• Gilead collaboration for three PRO-XTEN T-cell engagers · Vir Biotechnology

Verastem’s KRAS G12D inhibitor VS-7375 produced a 41% confirmed response rate in second-line or later pancreatic cancer at its selected 900 mg daily dose.

In the phase 1/2 TARGET-D 101 trial, 9 of 22 patients had confirmed objective responses, including one complete response. Another patient had an unconfirmed complete response. In second line specifically, 4 of 8 responded. Verastem plans to open a first-line phase 3 trial in metastatic pancreatic cancer by the end of 2026. It later priced a $250m stock and pre-funded warrant offering to fund clinical development and other corporate purposes.

• VS-7375 50% confirmed ORR in 2L pancreatic cancer and two CRs in 2L+ · Verastem

• Pricing of $250m common stock and pre-funded warrant offering · Verastem

Galderma agreed to buy ZO Skin Health in an all-cash transaction valuing the business at $1.5bn on an enterprise value basis.

ZO’s physician-dispensed skincare business generated ~$330m in net sales in the 12 months to June and sells through more than 16,500 physician, medical-spa and online accounts in over 40 countries. Closing is expected in Q4 2026/early 2027.

• To acquire ZO Skin Health, strengthening physician-dispensed skincare · Galderma

GSK told a Belgian works council it could cut up to 1,217 jobs.

The proposed cuts cover 482 office and 735 managerial posts across R&D, production and international support functions in Wallonia.

• Over 1,200 jobs under threat at vaccine manufacturer GSK in Belgium · VRT NWS

Senior dementia specialists asked Yvette Cooper for help getting Leqembi (lecanemab) and Kisunla (donanemab) onto the NHS, The Times reported.

The letter said both Alzheimer’s drugs are “genuine advances” that could substantially improve memory clinics’ capacity to help people with Alzheimer’s. The drugs treat the underlying cause of Alzheimer’s by clearing toxic amyloid protein from the brain. Trial data suggests that lecanemab slows memory decline by up to 27% over 18 months.

• Give breakthrough Alzheimer’s drugs on NHS, dementia experts say · The Times

AI drug developer Iambic Therapeutics priced its $150m Nasdaq IPO at $16.

It offered 9,375,000 shares, with trading beginning on 15 October. Iambic says its AI platform took lead candidate IAM1363 to the clinic in about two years.

• Iambic announces pricing of Initial Public Offering · Iambic

Thursday 15 October

OMass Therapeutics raised $310m to take OMS1620 through phase 2 in adrenal disorders.

Bain Capital Life Sciences, Forbion and Novo Holdings co-led the Series C in the University of Oxford spin-out, taking its total funding to about $475m. The money will advance OMS1620 through phase 2 in congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and support initial clinical data from OMass’s SLC15A4 programme. Syncona, its listed Series A investor, committed $10.1m.

• $310m Series C to advance endocrinology and immunology pipeline · OMass

• OMass raises $310m in Series C financing · Syncona

OrbiMed raised $3bn across two new funds, more than $1bn of it for Asia.

OrbiMed Private Investments X raised more than $1.9bn for US and European venture deals. OrbiMed Asia Partners VI raised more than $1bn for growth and venture investment across Asia.

• OrbiMed raises $3bn across global and Asia private equity funds · OrbiMed

AstraZeneca signed an AI-enabled drug discovery deal with insitro in liver disease and cardiomyopathies.

The AI company receives $40m upfront and up to about $1.46bn in milestones, plus single-digit royalties. The work covers metabolic dysfunction-associated steatohepatitis (MASH) and heart muscle disease.

• Strategic collaboration with insitro for cardiometabolic diseases · AstraZeneca

The EMA’s human medicines committee backed Rasonque for metastatic pancreatic cancer, following US approval in August.

The Committee for Medicinal Products for Human Use (CHMP) recommended EU approval of Revolution Medicines’ Rasonque (daraxonrasib), an oral RAS inhibitor, for adults whose disease has progressed after at least one treatment. In a 500-patient phase 3 trial, median survival was 12.4 months against 6.7 months on chemotherapy, and median progression-free survival was 7.2 months against 3.6 months. A phased review cut the assessment to fewer than 150 active review days. The European Commission now takes the final decision.

• EMA recommends authorisation of Rasonque for metastatic pancreatic cancer · EMA

Labcorp agreed to pay $722m in cash for selected assets of OPKO Health’s BioReference Health business, including the 4Kscore prostate cancer test.

Labcorp expects the acquired businesses to generate ~$250m in revenue in the first year after closing. Up to $150m more depends on 4Kscore sales over up to ten years. The assets include clinical, women’s health, urology and corrections testing, more than 80 patient service centres and 4Kscore. The deal needs US antitrust clearance.

• Agreement with OPKO Health to acquire select BioReference Health assets · Labcorp

The FDA approved Roche’s Enspryng for adults with thyroid eye disease.

Roche says Enspryng (satralizumab) is the first at-home subcutaneous treatment for the condition. The approval is based on two phase 3 SatraGO trials. SatraGO-2 met its primary endpoint, with a proptosis response in 52.9% of Enspryng patients against 23.4% on placebo among patients with active thyroid eye disease. SatraGO-1 missed its primary endpoint. Enspryng is given every four weeks during maintenance.

• FDA approves Enspryng first at-home SC treatment thyroid eye disease · Genentech

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• Halozyme blocked Keytruda SC in eight European countries.

• Nobel Prize awarded to optogenetics pioneers Deisseroth, Hegemann and Nagel.

• Biohub and partners committed $1.8bn for AI biology data.

• Lilly won first-line approval for Jaypirca in CLL.

• AbbVie and Genmab reported positive phase 3 Epkinly data in first-line DLBCL.

Browse by day:

• Weekend catch-up: UK dementia · Jaypirca · Novo · CSL-Alentis

• Monday: Shionogi · Epkinly · Vaxcyte · Nobel · AZ

• Tuesday: Option Care · Recordati · Forbion · ArriVent · Caribou

• Wednesday: Keytruda SC · Biohub · WHO · Cerevance

• Thursday: argenx · Viatris-Pacira · Isomorphic valuation · MSK

• Friday: TRexBio IPO

Weekend catch-up

UK Research and Innovation (UKRI) launched an £80m challenge to speed up dementia diagnosis in the NHS.

Nine technologies will be tested in NHS services, among them faster MRI scans, a remote cognitive assessment and a panel of blood biomarkers. Only those that prove they work get further money. The target is for 92% of people to be diagnosed within 18 weeks of referral, against about 60% today.

• £80m challenge for faster, better dementia diagnosis launched · UKRI

The FDA approved Lilly’s Jaypirca as a first-line treatment for chronic lymphocytic leukaemia (CLL).

Jaypirca (pirtobrutinib), a non-covalent BTK inhibitor, is now cleared for previously untreated CLL or small lymphocytic lymphoma without a known 17p deletion. In the phase 3 BRUIN CLL-313 trial against chemoimmunotherapy, the hazard ratio for progression-free survival (PFS) was 0.20. Median PFS was not reached, against 33.5 months.

• FDA approves pirtobrutinib for previously untreated CLL or SLL · FDA

The FDA extended its review of Novo Nordisk’s haemophilia A drug denecimig because of problems at a manufacturing site.

The agency has found no deficiencies in the clinical efficacy or safety data and has set no new timeline, Novo said. A decision had been expected in Q3 2026. Novo now aims to launch the bispecific antibody in the US in H1 2027.

• Update on the denecimig Biologics License Application in the US · Novo Nordisk

CSL agreed to pay Alentis Therapeutics $355m upfront to co-develop the kidney and liver drug lixudebart.

Lixudebart is an antibody against claudin-1. The companies agreed to split profits 55:45 in CSL’s favour, with up to $1.2bn for Alentis in commercial milestones. CSL will fund the phase 2 and planned phase 3 trials in ANCA-associated vasculitis with rapidly progressive glomerulonephritis, plus phase 2 trials in two other rare diseases.

• CSL & Alentis announce partnership to develop & commercialise lixudebart · CSL

Monday 5 October

Shionogi agreed to buy IntraBio for $2bn upfront, gaining the approved rare-disease drug Aqneursa.

Aqneursa (levacetylleucine) has been approved in the US since 2024 for Niemann-Pick disease type C, and the FDA cleared it for ataxia-telangiectasia on 18 September. The deal follows Shionogi’s purchase of global rights to Radicava (edaravone) in April.

• Shionogi continues rare disease expansion with acquisition of IntraBio · Shionogi

AbbVie and Genmab’s Epkinly improved on R-CHOP in first-line lymphoma.

In the phase 3 EPCORE DLBCL-2 trial in newly diagnosed diffuse large B-cell lymphoma (DLBCL), adding fixed-duration Epkinly (epcoritamab) to R-CHOP chemotherapy cut the risk of progression or death by 51% (HR 0.49). The figure held in both the primary population, with IPI scores of 3 to 5, and the wider 2-to-5 group. The partners call it the first phase 3 win on PFS for a bispecific antibody combination in first-line DLBCL. They will now discuss next steps with regulators.

• Epcoritamab with R-CHOP improves PFS in newly diagnosed DLBCL · AbbVie

Vaxcyte’s 31-valent pneumococcal vaccine met all its primary endpoints in a phase 3 trial in adults.

In OPUS-1, with 4,047 adults, VAX-31 matched Prevnar 20 (PCV20) on all 20 shared serotypes and Capvaxive (PCV21) on 17 of 19, under a stricter non-inferiority bar than past trials. Serotypes 3 and 12F missed that bar against Capvaxive but cleared the historical one. Vaxcyte plans a Biologics License Application (BLA) in H1 2028, following further phase 3 studies.

• Vaxcyte reports positive topline data from OPUS-1 phase 3 trial of VAX-31 · Vaxcyte

• VAX-31 OPUS-1 pivotal phase 3 adult trial: topline results · SEC EDGAR

The Nobel Prize in Physiology or Medicine went to Karl Deisseroth, Peter Hegemann and Georg Nagel for developing optogenetics.

Hegemann and Nagel discovered channelrhodopsin, a light-sensitive ion channel in single-celled algae that opens when illuminated, generating an electrical signal. In 2005, Deisseroth showed that introducing the gene into mammalian neurons allowed their activity to be controlled with pulses of light.

Optogenetics has transformed neuroscience by allowing researchers to activate or silence specific populations of neurons and establish causal links between neural circuits, behaviour and memory. Beyond widespread research use, the technology is being developed clinically to restore partial sight in people with retinal degeneration.

• Nobel Prize in Physiology or Medicine 2026 · NobelPrize.org

AstraZeneca opened an R&D centre in Kendall Square, Cambridge Massachusetts.

With AstraZeneca’s nearby genomic medicine site, the centre will house nearly 2,000 researchers. It is part of >$1bn AstraZeneca is investing in Massachusetts, which will expand its workforce there by >50%, within its $50bn US investment programme.

• AstraZeneca opens new global strategic R&D centre in Kendall Square · AstraZeneca

Tuesday 6 October

CD&R and McKesson agreed to buy Option Care Health for an enterprise value of $5.8bn.

The buyers will pay $32.05 a share for the US home and alternate-site infusion provider, a 37% premium to Monday’s close. CD&R will hold about 51%, and McKesson will invest about $1.4bn for the other 49%. The agreement sets out a framework for McKesson to buy CD&R’s stake later. Closing is expected in H1 2027.

• CD&R and McKesson sign agreement to acquire Option Care Health · SEC EDGAR

CVC and Groupe Bruxelles Lambert raised their offer for Recordati to €53 a share.

Their bid vehicle, Respighi BidCo, raised the price from €51.29, called it its “best and final offer“ and extended acceptances to 23 October. CVC already holds about 46.8% of the Italian drugmaker and wants to delist it. Activist Palliser Capital and Recordati’s four independent directors had opposed the original price.

• Increase in the offer consideration to €53.00 per share · Respighi BidCo

• CVC and GBL raise their bid for Recordati to 53 euros per share · Il Sole 24 Ore

Forbion raised €2.3bn across two funds, the largest fundraise in its history and one of the largest life sciences VC fundraisings globally in the past five years.

The Dutch firm’s new growth and venture funds can back about 30 companies, and its assets under management rise to about €7.5bn. Eli Lilly invested alongside MN, PGGM, KfW Capital and the Kauffman Foundation.

• Forbion raises €2.3 billion across two new funds · Forbion

ArriVent’s firmonertinib failed its phase 3 trial in first-line lung cancer with EGFR exon 20 insertions.

FURVENT missed its primary endpoint of PFS by blinded independent review. At the 240 mg dose, median PFS was 11.0 months against 9.5 months with chemotherapy (HR 0.75, p=0.0654). ArriVent reported benefits on secondary measures, including investigator-assessed PFS and response rate, and is reviewing the full data before deciding the drug’s future. ArriVent shares fell 47% on the news, reflecting the setback to its lead development programme.

• ArriVent announces program update from the phase 3 FURVENT trial · ArriVent

Caribou Biosciences decided to stop developing its allogeneic CAR-T therapies and to explore strategic alternatives.

Caribou plans to discontinue vispa-cel in lymphoma and CB-011 in myeloma and to substantially reduce its workforce, incurring $15-$19m of restructuring costs. Caribou shares fell by about 60% as investors assessed the implications of abandoning its development pipeline. The decision highlights the financing challenges facing allogeneic CAR-T, which promises lower manufacturing costs and faster treatment than patient-derived (autologous) therapies but faces questions over immune rejection, durability and commercial competitiveness.

• Caribou Biosciences Form 8-K on restructuring & strategic alternatives · SEC EDGAR

Wednesday 7 October

Halozyme won a Dutch injunction blocking Keytruda SC in eight European countries.

The Hague court found that Merck’s subcutaneous (SC) Keytruda infringes Halozyme’s MDASE patent EP622. The dispute centres on the hyaluronidase technology licensed from South Korea’s Alteogen, which enables SC delivery of pembrolizumab. Halozyme had previously granted BMS exclusive rights to its ENHANZE technology for PD-1 therapies, underpinning the rival SC Opdivo Qvantig, approved in 2024.

Keytruda SC is approved in the US, but the Dutch ruling and an earlier German injunction restrict its European rollout. Merck disputes the patent’s validity and is also facing US litigation. Intravenous Keytruda is unaffected.

• Halozyme wins injunction on Keytruda SC in multiple European markets · Halozyme

• Halozyme stymies SC Keytruda launch as Merck vows to fight · Fierce Pharma

Biohub and its partners committed $1.8bn to build AI-ready bio data.

Biohub, the DOE and NIH expanded the Virtual Biology Initiative, launched in April with $500m from Biohub. DOE will invest >$500m over five years in biological measurement, modelling and computing, while NIH will contribute datasets developed with >$500m of prior federal investment. Google DeepMind, Isomorphic Labs and Meta are investing $300m. The initiative aims to generate large-scale, standardised experimental data to train predictive models of living cells, potentially allowing researchers to simulate biological interventions before testing in the lab.

• AI-ready biological data: $1.8 billion global commitment · Biohub

The WHO issued its first global guidelines on childhood obesity, allowing medicines for some adolescents but not children under 10.

The guidelines recommend against medicines, bariatric surgery and weight-loss devices for children under 10. Adolescents aged 10-19 may receive medicines only after a supervised lifestyle programme has failed. Bariatric surgery may be considered for severe adolescent obesity. In 2024, 170m people aged 5-19 lived with obesity.

• WHO issues first global guidelines on child and adolescent obesity · WHO

Cerevance’s once-daily pill solengepras met the primary endpoint of a phase 3 trial in Parkinson’s disease.

In ARISE, solengepras was added to existing Parkinson’s treatments in 341 patients experiencing motor fluctuations. The 150 mg dose reduced daily OFF time by 0.61 hours more than placebo at week 12 (p=0.0350) and increased ON time without troublesome dyskinesia by 0.60 hours. The drug inhibits GPR6 rather than targeting dopamine receptors. Cerevance plans FDA discussions on a potential filing.

• Positive phase 3 ARISE results for once-daily oral solengepras · Cerevance

Thursday 8 October

argenx stopped its phase 3 UNITY trial of Vyvgart Hytrulo in Sjögren’s disease.

An independent data monitoring committee concluded that the trial was unlikely to meet its primary endpoint. argenx shares fell 12% on the news, reflecting the loss of a potentially significant expansion opportunity for Vyvgart.

• argenx provides update on phase 3 UNITY study in Sjögren’s disease · argenx

Viatris agreed to buy Pacira BioSciences at a $1.65bn equity value, adding two non-opioid pain products.

Viatris will pay $36.50 a share in cash for the maker of Exparel (bupivacaine liposome), for post-surgical pain, and Zilretta (extended-release triamcinolone acetonide), for osteoarthritis knee pain. Pacira had about $746m of revenue in the year to June. The deal should close by the end of 2026.

• Viatris agrees to acquire Pacira BioSciences · Viatris

Isomorphic Labs is in early talks to raise money at a valuation of $40bn to $50bn, Bloomberg reported.

The Alphabet-backed AI drug discovery company raised $2.1bn in May at an undisclosed valuation. The proposed valuation compares with a market capitalisation of about $2bn for Recursion Pharmaceuticals. Isomorphic has yet to advance its first drug into clinical trials.

• Isomorphic Labs in funding talks for at least $40 billion value · Bloomberg

• Isomorphic Labs reportedly raising at up to $50B valuation · SiliconANGLE

Memorial Sloan Kettering researchers found that restricting germline testing by age could miss most inherited cancer risk variants.

Testing 39,184 patients with solid tumours identified pathogenic germline variants in 16.3%. Restricting testing to patients under 50 would have missed 4,601 carriers, or 72% of those identified. The researchers, publishing in Cancer Discovery, argue for offering germline testing to all cancer patients regardless of age, with implications for treatment and screening of relatives.

• Current age-based genetic testing in patients with cancer may miss majority of inherited gene variants · AACR

Friday 9 October

TRexBio priced its IPO at $14 a share for expected proceeds of about $117m.

The company, which works on tissue regulatory T-cell biology, sold 8.3m shares. Trading on Nasdaq under TRXB was due to start on 9 October.

• TRexBio announces pricing of initial public offering · TRexBio

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• AstraZeneca and Summit agreed a $2bn equity deal.

• Lilly’s EloraTZP cut more weight than Zepbound in phase 2b.

• Sanofi and Regeneron widened their alliance for $1bn upfront.

• Novo and Hengrui agreed an obesity-pill licence worth up to $2.6bn.

• Novartis and Abogen signed a $575m-upfront mRNA deal.

Browse by day:

• Weekend catch-up: Mirum · Gazyva · Carvykti · BioWorld · AbbVie

• Monday: Merck · Kodiak · Roche-Chugai · Mirum · AZ-Summit · Egetis

• Tuesday: Novo-Hengrui · uniQure · PhRMA · Oura · Lilly retatrutide · BMS

• Wednesday: Merck · Lilly EloraTZP · US Medicare

• Thursday: Sanofi-Regeneron · Boehringer · Regeneron

• Friday: AZ-Summit Datroway · Novartis-Abogen

Weekend catch-up

Merck and Daiichi Sankyo withdrew their FDA application for a B7-H3 antibody-drug conjugate (ADC) in lung cancer.

The partners pulled the accelerated approval filing after the FDA said the data, including from the phase 2 IDeate-Lung01 trial, did not support it. Enrolment continues in the phase 3 IDeate-Lung02 trial.

• Ifinatamab Deruxtecan Biologics License Application for Certain Patients with Previously Treated Extensive-Stage Small Cell Lung Cancer Voluntarily Withdrawn · Daiichi Sankyo

The FDA approved Mirum’s Atebrioz for the ultra-rare bone disorder fibrodysplasia ossificans progressiva.

Atebrioz (zilurgisertib), an oral ALK2 inhibitor licensed from Incyte, is cleared for patients aged 12 and over. In the PROGRESS study, new heterotopic bone volume fell by 3.2 cm³ at week 24, against an increase of 24.6 cm³ on placebo. Mirum expects a US launch in October and puts annual revenue potential at >$200m.

• Mirum Pharmaceuticals Form 8-K on Atebrioz approval and AZURE-1 · SEC EDGAR

The FDA approved Roche’s Gazyva for frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome.

The indication covers patients aged two and over who are in complete remission. In the phase 3 INShore study, 95% of patients on Gazyva (obinutuzumab) were relapse-free after week 8 and in complete remission at one year, against 73% on mycophenolate mofetil. Genentech calls it the first FDA-approved treatment for the disease in 70 years. It is Gazyva’s second immune-mediated approval, after lupus nephritis.

• FDA approves Gazyva for idiopathic nephrotic syndrome · Genentech

Ten of 20 myeloma patients given one Carvykti infusion after one to three prior lines remained alive and progression-free at five years.

At the International Myeloma Society meeting in Glasgow, 10 of 20 patients treated with Carvykti (ciltacabtagene autoleucel) in CARTITUDE-2 Cohort A remained progression-free without maintenance at a median 60.7 months of follow-up, and 69.2% were alive at five years.

• Carvykti data shows half of patients progression-free at five years · Legend Biotech

BioWorld counted a record $199.2bn of biopharma deal value in the first eight months of 2026.

The January-August total was $186.5bn in 2025 and $111.9bn in 2019. May ($41.9bn) and June ($34.6bn) were among this year’s strongest months. BioWorld published figures on the med-tech sector, seeing $81.0bn of med-tech M&A value through August 2026, more than double a year earlier. The January-August total was $32.8bn in 2025. BioWorld puts the rebound down to fewer, bigger deals.

• Blockbusters lift 2026 biopharma deal value to record $199B · BioWorld

• Fewer, bigger deals drive med-tech M&A rebound in 2026 · BioWorld

Scrip found that AI-led companies took five of the ten largest biopharma venture rounds of 2026.

Recent financings included Isomorphic Labs’ $2.1bn Series B, Earendil Labs’ $787m raise, Chai Discovery’s $400m Series C and Enveda’s $311m Series E. The concentration points to investors backing AI-led companies at biotech scale, particularly those combining computational platforms with their own drug-development pipelines.

• Big money keeps flowing to AI-enabled drug discovery and development · Scrip

The FDA approved AbbVie’s Juvmo for Parkinson’s disease.

Juvmo (tavapadon) is the first selective D1/D5 receptor agonist approved for the disease, taken once daily with or without levodopa. In the phase 3 TEMPO-3 trial it increased daily on-time without troublesome dyskinesia by 1.7 hours, against 0.6 hours on placebo.

• US FDA approves AbbVie’s Juvmo (tavapadon) for Parkinson’s disease · AbbVie

Monday 28 September

Merck licensed a KRAS G12D inhibitor for $400m upfront.

SciBrunch Therapeutics granted worldwide rights to SPR2015, a preclinical oral molecular-glue inhibitor, in a deal worth up to $2.13bn with milestones. The transaction has closed.

• Merck Enters into Exclusive Global License Agreement with SciBrunch Therapeutics for SPR2015, an Investigational Oral KRAS G12D (ON) Inhibitor · Merck

Kodiak Sciences’ tarcocimab matched Eylea in a phase 3 trial in wet age-related macular degeneration.

In DAYBREAK, tarcocimab tedromer was non-inferior to Eylea (aflibercept) on vision gains at one year, and 54% of patients reached a six-month dosing interval under strict treat-to-dryness retreatment rules. Kodiak’s second candidate, tabirafusp alfa tedromer, also met its vision endpoint. Kodiak plans a three-indication filing for tarcocimab in Q4 2026.

• Zenkuda & tabirafusp-ted Meet Primary Endpoints in Pivotal Trial in wAMD · Kodiak

Roche is returning antibody emugrobart to Chugai after an interim obesity analysis.

An interim analysis of the phase 2 GYMINDA study, which paired the anti-myostatin antibody with GLP-1/GIP drugs, found clinically meaningful weight loss unlikely. Chugai is preparing to restart development in spinal muscular atrophy, which Roche dropped in March, and is weighing an out-licence.

• Notice regarding discontinuation of development of GYM329 for obesity · Chugai

Mirum’s brelovitug met the primary endpoint of a phase 3 trial in chronic hepatitis delta.

At week 24 of AZURE-1, 56% of patients on 300 mg weekly and 45% on 900 mg every four weeks reached combined viral response and ALT normalisation, against none on delayed treatment (p<0.0001). Mirum plans to file a Biologics License Application (BLA) in H1 2027 and puts annual revenue potential at >$1bn.

• Mirum Pharmaceuticals Form 8-K on Atebrioz approval and AZURE-1 · SEC EDGAR

AstraZeneca agreed to invest $2bn in Summit for about 12% of the company.

AstraZeneca is buying newly issued convertible preferred stock that gives it rights equivalent to about 12.0% of Summit’s common stock. The two will test Summit’s PD-1/VEGF bispecific ivonescimab with AstraZeneca’s Claudin 18.2 ADC sonesitatug vedotin in gastrointestinal cancers, each keeping the rights to its own drug. A memorandum of understanding points to a wider programme pairing ivonescimab with more AstraZeneca cancer drugs. Akeso, which engineered ivonescimab, keeps the rights in China and other regions.

• AstraZeneca announces strategic equity investment and clinical collaboration with Summit · AstraZeneca

The FDA approved Egetis Therapeutics’ Emcitate, the first US treatment for MCT8 deficiency.

Emcitate (tiratricol), a thyroid hormone receptor agonist, treats peripheral thyrotoxicosis in the X-linked disorder, in which median life expectancy is about 35 years. The Swedish company also received a rare paediatric disease priority review voucher, which it may sell in Q4 2026.

• FDA Approval of EMCITATE® for Patients with MCT8 Deficiency · Egetis Therapeutics

Tuesday 29 September

Novo Nordisk agreed to license Hengrui’s weekly obesity pill for up to $2.6bn.

Novo will pay $300m upfront for rights to HRS-1596 outside mainland China, Hong Kong, Macao and Taiwan. The phase 1-ready GLP-1/GIP dual agonist is designed for once-weekly oral dosing, and Hengrui has Chinese approval to start phase 1 trials in weight management and type 2 diabetes. The deal is expected to close in Q4 2026.

• Novo and Hengrui Pharma enter exclusive license agreement for HRS-1596 · Novo

uniQure’s Huntington’s disease gene therapy missed significance on its primary measure at four years.

In 12 high-dose patients at 48 months, AMT-130 slowed progression on the composite cUHDRS score by 44% against an external control (p=0.144), and on Total Functional Capacity by 61% (nominal p=0.008). uniQure says missing data in the updated control understate the effect. Its BLA is anchored on previously reported 36-month data. An updated analysis of 15 high-dose patients at that timepoint showed 80% slowing on cUHDRS.

• uniQure announces additional data from AMT-130 studies in Huntington’s disease · SEC EDGAR

PhRMA named former House Majority Leader Eric Cantor as its next president and chief executive.

Cantor, latterly vice chairman of Moelis & Company, starts on 9 November. He succeeds Stephen Ubl, who has led the US drug industry’s lobby since 2015.

• PhRMA selects Eric Cantor to lead organization as next president and CEO · PhRMA

Oura postponed its IPO of up to $2.2bn, citing uncertainty in the market.

The smart ring maker had planned to sell 50 million shares at $40 to $44. It has 5.7 million paying members and expects revenue to rise 90% in its 2026 financial year.

• Oura latest US IPO hopeful to delay listing as market jitters deepen · Reuters

• Oura Inc. Form S-1/A · SEC EDGAR

Lilly’s retatrutide cut weight by 20.8% over 80 weeks in obesity and type 2 diabetes.

At the European Association for the Study of Diabetes (EASD) meeting in Milan (published in The Lancet), 59.5% of patients on the 12 mg dose in the phase 3 TRIUMPH-2 trial no longer met the BMI criteria for obesity, and HbA1c fell by up to 1.6%. Lilly plans to file in Q1 2027.

• Triple agonist retatrutide delivered substantial weight loss & A1C reduction · Eli Lilly

Bristol Myers Squibb tightened liver monitoring in the long-term extension of its phase 3 admilparant trial after liver injuries and a trial participant’s death.

BMS submitted the amendment for European trial sites in May, classifying liver injury as a newly important potential risk. A spokesperson confirmed a “limited number” of liver events, including a death involving liver injury, but said it was unclear whether the patient received admilparant. Shares in BMS and Contineum, which has a similar drug, fell late on Tuesday. BMS expects results by the end of the year.

• BMS trial revisions shake confidence in new class of fibrosis drugs · BioPharma Dive

Wednesday 30 September

Merck detailed phase 2b tulisokibart results in hidradenitis suppurativa.

At week 16, 72% of patients on the top dose of the anti-TL1A antibody reached HiSCR50, and 41% reached HiSCR75 against 15% on placebo. Merck, which gained tulisokibart in its $10.8bn Prometheus acquisition, will take it into phase 3 in the skin disease. Merck first said the study had met its endpoints in its Q2 results on 4 August.

• Merck’s tulisokibart met primary & key secondary endpoints in phase 2b · Merck

• Merck announces second-quarter 2026 financial results · SEC EDGAR

Lilly’s EloraTZP cut more weight than Zepbound in type 2 diabetes.

In a 48-week phase 2b trial in 367 adults with obesity or overweight and type 2 diabetes, presented at EASD, the eloralintide and tirzepatide combination cut weight by up to 23.3% against 14.8% on Zepbound 15 mg. HbA1c fell by up to 2.9%, against 2.4%. Adverse events led 10.8% to 27.0% of patients on EloraTZP to stop treatment, against 2.9% on Zepbound, with gastrointestinal events most common. Lilly attributed the higher discontinuation rate partly to escalating doses of both drugs simultaneously and plans an optimised escalation schedule in phase 3.

• Lilly’s EloraTZP delivered greater weight loss & A1C reduction vs. tirzepatide · Eli Lilly

• Experimental Lilly combination drug tops Zepbound in weight-loss trial · Reuters

The Centers for Medicare and Medicaid Services (CMS) finalised its GLOBE drug-pricing demonstration for Medicare Part B.

GLOBE will test a new rebate formula for certain Part B drugs against prices in economically comparable countries. The model starts on 1 January 2027, with a five-year performance period from 1 April 2027. CMS now projects $440m of Medicare Part B savings over the seven-year payment period, down from $11.9bn in the proposed rule.

• CMS Finalizes New Mandatory Drug Payment Model to Deliver Lower Prices · CMS

• Global Benchmark for Efficient Drug Pricing (GLOBE) final rule · Federal Register

Thursday 1 October

Sanofi to pay Regeneron $1bn upfront to widen their antibody alliance.

The Dupixent (dupilumab) partners will co-develop four long-acting Regeneron antibodies, against IL-13, IL-4 and IL-4Rα plus an IL-4xIL-13 bispecific, with up to $7bn more in milestones. The two will share development and commercialisation costs and split future profits 50:50, and the Dupixent profit share is unchanged. Only the IL-13 antibody REGN20423 is in the clinic, in phase 1 for atopic dermatitis. Regeneron leads research and development; Sanofi leads global commercialisation.

• Sanofi & Regeneron expand alliance with long-acting antibodies · SEC EDGAR

Boehringer Ingelheim’s survodutide cut weight by up to 13.1% in a phase 3 trial in obesity and type 2 diabetes.

In SYNCHRONIZE-2, 752 treated adults lost up to 13.1% at 76 weeks against 3.1% on placebo, on the efficacy estimand. HbA1c fell by up to 1.21%. Gastrointestinal side effects led 18% of patients on survodutide to stop treatment, against 1.2% on placebo. Boehringer said discontinuations usually occurred during dose escalation and later trials allow more flexible titration.

• Survodutide achieves up to 13.1% weight loss in Phase III · Boehringer Ingelheim

• Survodutide Once Weekly in Adults with Obesity and Type 2 Diabetes · NEJM

Regeneron’s trevogrumab halved lean-mass loss as an add-on to Wegovy in a phase 2 trial.

In COURAGE, adding 75 mg of trevogrumab to Wegovy (semaglutide) 2.4 mg reduced lean-mass loss by 50.7% relative to Wegovy alone at 26 weeks, without meaningfully adding to weight loss. In an MRI substudy it prevented about 70% of thigh-muscle loss. Regeneron plans a phase 2 in adults with obesity and low muscle mass.

• COURAGE trial confirms trevogrumab prevents lean mass & muscle loss · Regeneron

Friday 2 October

AstraZeneca and Daiichi Sankyo agreed a clinical collaboration with Summit to test Datroway with ivonescimab.

The companies will evaluate the combination across multiple tumour types, including lung and breast cancers, beginning with a phase 3 trial in first-line triple-negative breast cancer. Each will retain development and commercial rights to its own drug and share trial costs.

• AstraZeneca and Daiichi announce clinical collaboration with Summit · AstraZeneca

Novartis agreed to pay Abogen $575m upfront for an mRNA-encoded T-cell engager.

China’s Abogen Biosciences has agreed to grant worldwide rights to ABO2203, a CD19xCD3 T-cell engager for autoimmune disease that is encoded in mRNA so the body makes the engager itself. Novartis also gets options on other programmes from Abogen’s RNA platform. If all options are exercised, milestones could reach $7.2bn.

• Licensing and option agreement with Novartis · Abogen Biosciences

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• An FDA panel backed Grail’s Galleri cancer test.

• Telix agreed to buy ITM for $1.65bn upfront.

• The White House said all 50 state Medicaid programmes will get MFN drug prices.

• Nine European pharmas urged action to reverse Europe’s declining drug research.

• The FDA approved Lilly’s once-weekly insulin for type 2 diabetes.

Browse by day:

• Weekend: Medicaid prices · MFN deals · China deals · Inluriyo · NIH · IntraBio

• Monday: Telix-ITM · Beacon · Novo · Iambic

• Tuesday: Celldex · Roche obesity · Amgen · Otsuka-Ionis · EU pharma letter

• Wednesday: Roche kidney · Basecamp · Enveda · Anthropic · Grail

• Thursday: Lilly-InnoCare · Lilly insulin · Merck · Kobayashi · Novo-Nanexa · ADARx

Weekend catch-up

All 50 US state Medicaid programmes will get most-favoured-nation drug prices, the White House said on Friday.

Under the GENEROUS model, participating manufacturers pay rebates on covered brand-name drugs so that Medicaid’s final price does not exceed the MFN price. The Council of Economic Advisers puts the saving at $36.6bn to the federal government and $27.6bn to states over a decade.

• Lower drug prices for all 50 state Medicaid programs · The White House

Public Citizen published heavily redacted versions of Pfizer’s and Lilly’s MFN agreements, obtained through freedom-of-information requests.

According to its analysis, Pfizer’s agreement provides for sharing with the US government some additional revenue from higher drug prices abroad. Lilly’s allows a country’s price to be excluded from the MFN calculation if it stops supplying the drug there. Its Mounjaro and Zepbound products are excluded from the GENEROUS Medicaid pricing programme and covered by a separate arrangement.

• FOIA reveals pharma, not patients, are favored in drug pricing deals · Public Citizen

US Treasury rules could preserve most drug licensing deals with China, Reuters reported.

Restrictions would focus on pathogens and biotechnology that could be weaponised, according to sources briefed on the process. The rules have not been finalised. The Wall Street Journal also reported that drugmakers were lobbying the administration to protect their ability to license Chinese drugs.

• US weighs allowing most pharma licensing deals with China · Reuters

• Pharmaceutical companies and the administration’s China policy · The Wall Street Journal

The FDA approved Lilly’s Inluriyo with Verzenio for ESR1-mutated advanced breast cancer.

The approval covers adults with ER-positive, HER2-negative disease that progressed after at least one line of endocrine therapy. In EMBER-3’s ESR1-mutated subgroup, the oral SERD Inluriyo (imlunestrant) plus Verzenio (abemaciclib) doubled median progression-free survival to 11.1 months, versus 5.5 months with Inluriyo alone.

• FDA approves imlunestrant for ESR1-mutated advanced breast cancer · FDA

A White House plan to give political appointees veto power over NIH grants stalled after bipartisan opposition.

The proposed executive order would add political review after scientific assessment of grant applications. Senate Appropriations Committee chair Susan Collins wrote to budget director Russ Vought and NIH director Jay Bhattacharya on Tuesday urging them not to proceed. By Wednesday, the plan was “looking dead”, STAT reported.

• Sen. Collins warns not to proceed with NIH order · Senate Appopriations Committee

• White House move on NIH grants stalled by bipartisan pushback · STAT

The FDA approved IntraBio’s Aqneursa for ataxia-telangiectasia.

Aqneursa (levacetylleucine) was cleared for ataxia in patients weighing ≥15 kg, on a crossover trial in 73 patients aged 4 to 50. IntraBio says it is the first drug approved for the disease anywhere. It was already approved in Niemann-Pick disease type C.

• US FDA approval of AQNEURSA for ataxia-telangiectasia · IntraBio

Monday 21 September

Telix agreed to buy isotope maker ITM for $1.65bn upfront.

The deal includes $1.25bn in Telix shares and $302m in net debt, with up to $700m tied to approvals and sales of ITM-11, ITM’s lutetium-177 treatment for neuroendocrine tumours. ITM had $273m in sales in 2025. The acquisition strengthens isotope supply for Telix’s pipeline and adds ITM’s development assets. ITM plans to resubmit ITM-11 after an FDA rejection in August over manufacturing and third-party facility issues. According to Telix, the FDA identified no safety or efficacy deficiencies.

• Telix and ITM join forces to create a radiopharmaceutical powerhouse · Telix Pharma

• Acquisition investor presentation · Telix / SEC EDGAR

Beacon’s eye gene therapy met its primary endpoint in a pivotal phase 2/3 trial.

Laru-zova met the FDA-endorsed primary endpoint in VISTA, an 85-patient trial in males with X-linked retinitis pigmentosa (XLRP), an inherited disease causing progressive vision loss. At 12 months, 31.0% of the high-dose group and 24.1% of the low-dose group gained at least 15 letters on a low-light visual acuity test, compared with none in the untreated group. Beacon plans to begin a rolling US approval application later this year. UK investor Syncona owns 38.4% of Beacon. Its stake was valued at £183.4m at 30 June, before these results.

• Positive topline data from the pivotal VISTA trial of laru-zova · Beacon Therapeutics

• Beacon delivers positive topline PII/III results · Syncona

Novo’s shares fell 8% as its new strategy failed to reassure investors.

At its capital markets day in London, Novo targeted over five blockbuster launches by 2030 and over DKK150bn in pipeline sales in 2035. Chief executive Mike Doustdar acknowledged that management had to convince investors there was a bright future for Novo. Novo’s shares fell nearly 11% over the week to Thursday’s close, wiping approximately $20bn off its market value. Positive phase 3 results for CagriSema, its combination of cagrilintide and semaglutide, were not enough to lift sentiment.

• Highlights to be presented at Novo’s Capital Markets Day 2026 · Novo

• CagriSema delivers superior weight loss versus tirzepatide in REIMAGINE 5 · Novo

• Novo struggles to appease restless investors with new strategy · Fierce Biotech

AI drug developer Iambic Therapeutics filed for a Nasdaq IPO and announced an AbbVie discovery partnership.

Its lead candidate, IAM1363, is an oral, brain-penetrant HER2 inhibitor in phase 1/1b, with a registrational trial potentially starting in 2027. The multi-year AbbVie collaboration will apply Iambic’s AI platform to small-molecule discovery in immunology, neuroscience and oncology. Iambic will receive an upfront payment and could earn milestones and royalties; amounts were not disclosed.

• Iambic Therapeutics Form S-1 · SEC EDGAR

• Iambic collaboration to accelerate AI-driven drug discovery · AbbVie

Tuesday 22 September

Celldex’s barzolvolimab met the primary endpoint in two phase 3 trials in chronic spontaneous urticaria.

The anti-KIT antibody met all key secondary endpoints in EMBARQ-CSU1 and CSU2, which enrolled 1,939 patients whose symptoms were inadequately controlled by antihistamines. Across both trials and doses, 42–46% had no itch or hives at week 12, versus 9–13% on placebo. Benefits were also seen in patients who had not responded to omalizumab. Celldex plans to file for FDA approval in 2027.

• Positive results from phase 3 EMBARQ-CSU1 and EMBARQ-CSU2 · Celldex

Roche’s enicepatide produced 15.5% weight loss in a phase 2 diabetes trial.

Enicepatide (formerly CT-388), its once-weekly GLP-1/GIP receptor agonist, produced 15.5% weight loss and reduced HbA1c by 2.65 percentage points at the highest dose in a 447-patient diabetes trial. The 15.5% weight loss at 48 weeks puts Roche’s candidate in the same range as Lilly’s tirzepatide results in patients with diabetes, achieved over 72 weeks. Weight loss had not plateaued, although the separate trials do not establish comparative superiority. Phase 3 obesity trials are already underway.

• Roche announces positive phase II results for enicepatide in type 2 diabetes · Roche

Amgen’s dazodalibep met its phase 3 primary endpoint in Sjögren’s disease.

The CD40L-blocking fusion protein reduced systemic disease activity versus placebo at 48 weeks in OASIZ 301, which enrolled c.620 patients. Sjögren’s is an autoimmune disease that can cause debilitating dryness, fatigue, pain and organ damage, with no FDA-approved systemic treatment. Amgen has yet to release detailed results. A second phase 3 study, focused on patients with substantial symptoms but lower systemic disease activity, is expected to complete in Q4.

• Positive topline phase 3 results for dazodalibep in Sjögren’s disease · Amgen

Otsuka and Ionis reported the first positive phase 3 trial in FUS-ALS.

Ulefnersen, an RNA-targeted drug designed to reduce production of the FUS protein, met the primary endpoint in FUSION. It improved a combined measure of survival, rescue treatment and function versus placebo over 505 days (p=0.0005). FUS-ALS is a rare fast-progressing genetic form of motor neurone disease with no approved treatment targeting its underlying cause. The partners plan to discuss expedited regulatory submission routes with the FDA.

• Otsuka announces transformative phase 3 FUSION results for ulefnersen · Otsuka

Nine European pharma chairs called for action to reverse Europe’s declining share of drug research.

The chairs of companies including AstraZeneca, GSK, Novartis and Roche urged European governments to increase investment in medicines, accelerate clinical trials and speed up access to new treatments. Their joint letter said Europe’s share of global pharmaceutical R&D had fallen from 43% in 1990 to 31%, while its share of commercial clinical trials had halved over the past decade to 9%.

• Europe is losing the pharma investment race: an open letter · Novartis

Wednesday 23 September

Roche’s sefaxersen met its phase 3 primary endpoint in kidney disease.

The monthly injectable RNA-targeted therapy, licensed from Ionis, significantly reduced protein in the urine versus placebo at 37 weeks in IgA nephropathy, a progressive autoimmune kidney disease. Sefaxersen reduces production of complement factor B, which contributes to kidney damage.

• Positive interim data for sefaxersen in IgA nephropathy · Roche

London’s Basecamp Research raised a $140m Series C to develop bio AI models.

S32 led, with backing from NVIDIA and Menlo Ventures. Basecamp collects genomic data through biodiversity partnerships in >30 countries to train its EDEN models. The funding will support further model development and the company’s ambition to translate AI-designed DNA sequences into therapies. Its therapeutic work is preclinical.

• Basecamp raises $140M to advance AI-designed therapeutics · Basecamp Research

Enveda raised $311m to advance medicines derived from natural compounds.

Catalio Capital Management led the Series E, joined by investors including T. Rowe Price, Citadel’s Surveyor Capital, ICONIQ and existing backer Baillie Gifford. The round takes total funding above $845m. Enveda uses AI to identify promising natural compounds and medicinal chemistry to develop them into oral drugs. Funding will advance three clinical programmes spanning eczema, metabolic disease and inflammatory bowel disease into mid- and later-phase trials, advance further candidates into the clinic and expand the discovery platform.

• Enveda raises $311 million from leading AI and biotech investors · Enveda

Anthropic introduced its biology lab and reported an AI-assisted finding.

The Claude developer’s new Bay Area laboratory pairs AI analysis of genomic data with experiments conducted by human scientists. Its first reported findings concern a previously uncharacterised system of DNA repeats and an accessory gene alongside a known reverse transcriptase. Initial experiments showed that the repeats produce short RNAs, but the system’s function remains unknown. Independent commentary highlighted Claude’s ability to pursue promising leads, while noting that the study does not establish an advantage over conventional bioinformatics methods.

• Anthropic introduces its laboratory and initial findings · Anthropic

• Autonomous AI agents discover RTs with tandem repeat arrays · Preprint (PDF)

• An AI model found a new CRISPR-like biological system · The Conversation

An FDA panel backed Grail’s Galleri cancer test.

The advisory panel voted 7-2, with one abstention, that the blood test’s benefits outweigh its risks for screening adults aged 50 and older. Members unanimously supported its safety but split 6-4 on effectiveness. The panel questioned whether an “early detection” claim was supported, with some favourable votes conditional on revised labelling and further studies. Long-term benefits remain unestablished. The vote is non-binding, with an FDA decision expected in the coming months.

• FDA advisory committee votes in favor of approval of GRAIL’s Galleri test · GRAIL

• Galleri advisory committee meeting and supporting materials, 23 September · FDA

• Galleri advisory committee voting results and discussion summary · FDA

Thursday 24 September

Lilly signed a discovery deal with China’s InnoCare worth up to $3.35bn.

InnoCare will discover and advance compounds under an agreement offering up to $100m in upfront and near-term payments, plus approximately $3.25bn in potential development and commercial milestones. It could also receive tiered single-digit royalties on sales. The targets and therapeutic areas were not disclosed.

• Strategic research collaboration and license agreement with Lilly · InnoCare

The FDA approved Lilly’s Onswik, a once-weekly basal insulin for type 2 diabetes.

Onswik (insulin efsitora alfa) reduces basal insulin injections from 365 to approximately 52 a year. Approval was supported by four phase 3 QWINT trials involving more than 3,400 adults, which showed comparable HbA1c reductions to daily basal insulins. Lilly expects US pharmacy availability in the coming months. It follows Novo’s Awiqli, approved in March as the first weekly insulin in the US.

• FDA approves Lilly’s Onswik, a once-weekly basal insulin · Eli Lilly

• FDA approves once-weekly insulin for type 2 diabetes · FDA

Merck’s remigromig matched Lucentis in a pivotal diabetic macular oedema trial.

In the 984-patient BRUNELLO trial in diabetic macular oedema, both doses were non-inferior to Lucentis on improvement in vision at 52 weeks. The antibody activates the Wnt pathway, which helps maintain the blood-retinal barrier. However, proliferative diabetic retinopathy, vitreous haemorrhage and discontinuations due to adverse events were more frequent with remigromig. Merck is investigating these findings and will present detailed results in October. A second phase 2b/3 trial is ongoing.

• Remigromig met primary endpoint in pivotal phase 2b/3 BRUNELLO study · Merck

Japan’s Kobayashi Pharma received a take-private approach from NSSK and CVC.

The Japanese consumer-health group confirmed a preliminary, non-binding approach from affiliates of private equity firms NSSK and CVC Capital Partners. Reporting on Thursday put a potential deal at more than ¥500bn ($3.2bn), with possible participation by the founding family. Kobayashi is still recovering from a 2024 scandal involving contaminated red yeast rice supplements linked to kidney damage, and faces pressure from activist shareholder Oasis Management. No deal has been agreed.

• Kobayashi buyout talks report · Bloomberg

• Japanese, UK funds propose taking Kobayashi Pharma private · Nippon.com

• Statement on take-private proposal (Japanese) · Kobayashi Pharmaceutical

Novo licensed Nanexa’s technology to develop monthly and quarterly injections.

The agreement covers up to five peptide programmes in obesity, type 2 diabetes and other cardiometabolic diseases. Swedish biotech Nanexa’s PharmaShell platform coats drug particles with an ultrathin shell to control their release, potentially extending the interval between injections. Novo will lead development and commercialisation. Nanexa could receive up to €1.165bn plus royalties, including €615m in combined upfront, development and regulatory payments (upfront not disclosed).

• Nanexa and Novo enter €1.165bn license for long-acting injectables · Nanexa

ADARx priced an upsized $446.3m IPO at the top of its range.

The siRNA drug developer priced 26.25m shares at $17, increasing the offering from approximately 21.9m shares. A concurrent investment by partner AbbVie brings expected combined gross proceeds to $535.2m and would give AbbVie a 4.9% stake. ADARx develops RNA-targeted medicines designed to reduce production of disease-driving proteins. Nasdaq trading under ADRX was scheduled to begin on Friday.

• ADARx announces pricing of upsized $446.3 million IPO · ADARx

• ADARx Pharmaceuticals Form S-1/A · SEC EDGAR

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• Akeso’s ivonescimab beat Keytruda on overall survival in lung cancer.

• NICE recommended Enhertu for NHS use in metastatic breast cancer.

• The FDA approved Curium’s Bexlutry, the first Lutathera equivalent.

• GSK and Hansoh’s ADC nearly doubled survival in small cell lung cancer.

• Scholar Rock won its first FDA approval, for the first SMA drug to target muscle.

Browse by day:

• Weekend catch-up: Akeso · GSK-Hansoh · Scholar Rock · AZ Etcamah

• Monday: Enhertu lung data · Curium · Sanofi · US FDA psychedelics

• Tuesday: GSK-Chimagen · Vera · US FDA trial pilot · Mayo-Thermo Fisher

• Wednesday: Novo-Anthropic · Sling · China ByteDance

• Thursday: UK NICE Enhertu · Ultragenyx · Bayer

Weekend catch-up

AstraZeneca’s Etcamah missed its primary endpoint in untreated advanced breast cancer.

On Friday evening AstraZeneca said the phase 3 SERENA-4 trial of Etcamah (camizestrant) with Ibrance (palbociclib) failed to beat an aromatase inhibitor plus Ibrance on progression-free survival in 1,371 patients with oestrogen receptor-positive, HER2-negative advanced breast cancer who had not been treated for advanced disease. The oral selective oestrogen receptor degrader stays approved in the US, EU and Japan with a CDK4/6 inhibitor for patients whose tumours develop an ESR1 mutation during first-line endocrine therapy, on the SERENA-6 trial.

• Update on SERENA-4 Phase III trial of Etcamah · AstraZeneca

Akeso’s ivonescimab beat Keytruda on overall survival.

In the phase 3 HARMONi-2 trial in China, ivonescimab monotherapy extended median overall survival to 30.8 months against 22.6 months for Keytruda (pembrolizumab) in first-line PD-L1-positive advanced non-small cell lung cancer (hazard ratio 0.73, p=0.009). Follow-up was 36 months at the 20 August cut-off. The benefit was largest in tumours with PD-L1 expression of 50% or more, where the hazard ratio was 0.58.

HARMONi-2 had already met its progression-free survival endpoint in 2024, which won the drug its Chinese approval. Survival is the harder test, and no drug had cleared it against Keytruda in this setting before. Akeso’s partner Summit Therapeutics holds rights outside China and is running its own phase 3 programme.

• Positive Overall Survival Results from HARMONi-2 · Akeso

GSK and Hansoh’s B7-H3 drug nearly doubled survival in relapsed small cell lung cancer.

In the phase 3 ARTEMIS-008 interim analysis, presented at the World Conference on Lung Cancer in Seoul, risvutatug rezetecan lifted median overall survival to 18.5 months against 10.3 months for topotecan. Hansoh calls it the first randomised phase 3 study anywhere to show a survival benefit for a B7-H3 antibody-drug conjugate in the disease. GSK holds rights outside mainland China, Hong Kong, Macau and Taiwan.

• Interim Results from Phase 3 ARTEMIS-008 Study · Hansoh Pharma

Roche and MediLink’s rival B7-H3 drug cut the risk of death by 54% in the same disease.

The phase 3 TAISHAN-302 trial in China gave Tam-Peli (tambotatug pelitecan) a median overall survival of 13.3 months against 9.4 months for topotecan, with median progression-free survival of 7.4 months against 2.8 and a response rate of 59.1% against 9.7%. Roche licensed the drug from MediLink in January and plans to start global phase 3 trials.

• Roche’s collaborator MediLink announces phase III data for Tam-Peli · Roche

Scholar Rock won FDA approval for the first muscle-targeted SMA treatment.

Isembyld (apitegromab) was cleared for adults and children aged two and over with spinal muscular atrophy who are already on a treatment targeting the SMN2 gene. All existing therapies target the motor neuron, whereas this drug acts directly on muscle. It is the first to demonstrate improved motor function on top of those treatments. In the phase 3 SAPPHIRE trial, Isembyld produced a 2.2-point improvement on the Hammersmith Functional Motor Scale-Expanded after a year, in the main efficacy population of 103 children aged 2 to 12. Scholar Rock also collected a priority review voucher.

• Scholar Rock Announces FDA Approval of ISEMBYLD · Scholar Rock

Monday 14 September

AstraZeneca and Daiichi Sankyo’s Enhertu added six months of progression-free survival in first-line HER2-mutant lung cancer.

In the phase 3 DESTINY-Lung04 trial, Enhertu reached a median of 14.3 months against 8.3 months for Keytruda plus chemotherapy in 454 patients, cutting the risk of progression or death by 37% (hazard ratio 0.63, p<0.0001). Response rate was 70.0% against 44.5%. At 46.9% maturity there was no observed overall survival benefit, and AstraZeneca pointed to imbalanced use of later therapies. Interstitial lung disease or pneumonitis occurred in 20.8% of Enhertu patients, with four Grade 5 events.

• Enhertu median PFS of 14.3 months in DESTINY-Lung04 · AstraZeneca

The FDA approved the first radioligand equivalent for GEP-NETs.

Curium’s Bexlutry (lutetium Lu 177 dotatate) was cleared for adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumours. It went through the 505(b)(2) pathway against Novartis’s Lutathera, supported by published evidence and bridging data showing a similar biological and chemical profile.

• Curium announces FDA approval of BEXLUTRY · Curium

Sanofi proposed handing 20 older medicines and three plants to Cheplapharm for a 26.4% stake.

The German family-owned group would take over products including Lovenox/Clexane (enoxaparin), excluding the US, and manufacturing sites in Hungary, Singapore and France. The portfolio transfer is planned to begin in Q1 2027 and the deal is expected to complete by Q3 2027, subject to employee consultation, regulatory approvals and customary closing conditions. Sanofi takes equity rather than cash, and says the deal will not change its 2026 guidance.

• Sanofi and Cheplapharm to create strategic partnership · Sanofi

Electra Therapeutics set IPO terms targeting $296.6m net at the midpoint.

The company filed to sell 21.67m shares at $14 to $16 on Nasdaq under the ticker ETRA, which would raise $296.6m net at the midpoint and $341.9m if underwriters take their full option. Lead candidate ipsoprubart is a pan-SIRP antibody in a registrational programme for secondary haemophagocytic lymphohistiocytosis.

• Electra Therapeutics, Inc. Form S-1/A · SEC EDGAR

Novo Nordisk will be known as Novo day to day.

The company rebranded around the single name Novo and the line “Lasting Health Starts Now”, and set out a new internal culture code. Novo Nordisk A/S remains the legal company name. A fuller strategy update comes at its Capital Markets Day in London on 21 September.

• Novo Nordisk enters next chapter · Novo Nordisk

Cellectis dropped its two allogeneic CAR-T programmes to become an in vivo gene editing company.

The board approved the change on 11 September. Cellectis will drop lasme-cel and eti-cel and look for partners for them, and put its money behind .HEAL-101, an in vivo base editor targeting APOC3 in severe hypertriglyceridaemia, and .HEAL-201, an epigenetic editor targeting PCSK9 in severe hypercholesterolaemia. It keeps its existing cell therapy partnerships with AstraZeneca, Allogene, Servier and Iovance, and the combined actions are designed to extend its cash runway into H2 2028.

• Cellectis Transformation To In Vivo Gene Editing · Cellectis

The FDA held a public hearing on how psychedelic medicines should be used.

The agency asked for views on the therapeutic use of psychedelics in supervised and supportive settings, with a first approval in the class now close. Access dominated: who would be allowed to administer such a drug, and how that would be credentialled. Clinical psychologist Geoff Bathje told the hearing he had been “disappointed to see the FDA minimize the importance of the therapeutic relationship in psychedelic research for the sake of isolating drug effects in clinical trials“.

• Future Therapeutic Use of Psychedelic Drugs Public Hearing · FDA

• FDA holds psychedelic hearing as historic approval looms · Fierce Biotech

Definium’s LSD therapy won a third phase 3 trial in 12 weeks.

In the 245-patient Panorama study, DT120 cut Hamilton Anxiety Rating Scale scores by 9.8 points at week 12 against 4.7 for placebo. Definium has a pre-NDA meeting in Q4 2026, with a filing planned for H1 2027, having also won in anxiety in the Voyage trial and in major depressive disorder in June.

• Topline Results from Phase 3 Panorama Study · Definium Therapeutics

Tuesday 15 September

GSK will pay up to $750m for a Chimagen trispecific T-cell engager in multiple myeloma.

GSK would acquire full global rights on closing for an upfront fee, with the rest tied to development and commercial milestones, and expects to start phase 1 trials in 2027. The agreement is subject to customary closing conditions. It is the second asset GSK has taken from the Chinese biotech, after CMG1A46, a CD19 and CD20 dual-targeted engager already in phase 1.

• GSK to acquire T cell-engager for multiple myeloma from Chimagen · GSK

Vera Therapeutics’ Trutakna held kidney function steady for two years in IgA nephropathy.

The final efficacy analysis of the phase 3 ORIGIN 3 trial, in 428 patients, met every prespecified endpoint. Trial investigator Richard Lafayette said the result showed “a significant reduction in risk of composite kidney disease progression, true stabilization of eGFR, and a favorable safety profile through two years“. Trutakna holds accelerated approval on proteinuria reduction. Vera plans to file for full approval in Q4 2026.

• TRUTAKNA Stabilized eGFR Through Two Years in ORIGIN 3 · Vera Therapeutics

The FDA opened applications for a pilot meant to speed up first-in-human trials.

The Expedited Investigational New Drug (IND) Pilot pairs drug sponsors with qualified research institutions and lets the agency review parts of an application on a rolling basis before the full filing. The FDA says first-in-human trials can take up to two years to complete in the US against much faster timelines in China and Australia. Applications close on 30 October, and 8 to 10 sponsor pairs will be chosen.

• FDA Launches Expedited IND Pilot, Begins Accepting Applications · FDA

Mayo Clinic and Thermo Fisher Scientific launched Precure to map disease before symptoms appear.

The new company will build a biomedical dataset out of one of the largest human multi-omics data-generation efforts to date, joining proteomic, genomic and longitudinal clinical data. Mayo brings biospecimens and longitudinal de-identified clinical data, Thermo Fisher the proteomics platforms.

• Mayo Clinic and Thermo Fisher Scientific launch Precure, LLC · Mayo Clinic

Wednesday 16 September

Novo Nordisk signed an AI discovery deal with Anthropic.

Novo will test Claude Science on specific research and development workflows, starting with scientific problems where it thinks the two companies together can do most, and will use Anthropic’s frontier models for AI-driven software development.

Anthropic chief executive Dario Amodei said increasing AI capability “brings with it the potential to compress a century’s worth of biological and medical breakthroughs into a decade“. No financial terms were given.

• Novo & Anthropic will collaborate to advance drug discovery with Claude · Novo

Sling Therapeutics raised $123m to advance an oral thyroid eye disease drug in phase 3.

Forbion led the series C, with TPG Life Sciences Innovations and Sectoral Asset Management taking part. Linsitinib is an oral IGF-1R inhibitor aiming to compete against the injectable drugs used now, and its global phase 3 ORBIT trial has started.

• Sling Therapeutics Announces $123 Million Series C Financing · Sling Therapeutics

ByteDance’s AI drug discovery unit closed a $290m round.

Reuters reported the first outside funding for Anew Labs since its spin-off from the Chinese technology group.

• ByteDance completes $290 million fundraising for AI drug unit · Reuters

Thursday 17 September

NICE recommended Enhertu for NHS use in final draft guidance.

Around 1,000 people a year with HER2-low advanced or metastatic breast cancer whose disease has progressed can now be offered Enhertu on the NHS. It had been the only breast cancer drug NICE could not recommend since 2018, with 25 others recommended in that period. NICE said a solution was agreed “following changes made to NICE methods as part of the UK/US Pharmaceutical Pricing Agreement“.

• NICE recommends Enhertu for people with HER2-low advanced breast cancer · NICE

• Arrangement between the US and the UK on pharmaceutical pricing · GOV.UK

The FDA approved Ultragenyx’s Fayuvi, the first treatment for Sanfilippo syndrome type A.

The one-time AAV gene therapy is cleared for the neurological effects of mucopolysaccharidosis type IIIA in children whose neurodevelopment is still preserved, on the Transpher A registration trial and follow-up running to nearly eight years. In the modified intention-to-treat population, Fayuvi produced a 23.5-point higher Bayley-III cognitive score than natural history. The FDA had refused the application in July 2025 over manufacturing controls and facility inspection findings. It is the company’s sixth approval, and came with a priority review voucher.

• Ultragenyx Announces Approval of FAYUVI Gene Therapy · Ultragenyx

• Ultragenyx Receives Complete Response Letter for UX111 · Ultragenyx

Bayer’s Kerendia won the first new US approval in 30 years for kidney disease in type 1 diabetes.

The FDA cleared Kerendia (finerenone) under priority review to reduce urinary albumin-to-creatinine ratio in adults with chronic kidney disease associated with type 1 diabetes, a reduction expected to lower the risk of sustained kidney function decline and end-stage kidney disease. The FINE-ONE results let Bayer bridge Kerendia’s outcomes evidence from type 2 diabetes. It is the drug’s third indication.

• KERENDIA Receives FDA Approval for CKD and Type 1 Diabetes · Bayer

Two more private biotechs lined up reverse mergers onto Nasdaq.

Lisata Therapeutics acquired Marea Therapeutics and agreed a concurrent private placement expected to raise about $225m, picking up MAR001/005 in severe hypertriglyceridaemia, now in phase 2b, and MAR002 in acromegaly. Separately, Aethlon Medical signed a definitive merger agreement with North Immunology, subject to closing conditions, alongside an oversubscribed $180m placement funding NOR-101, an IL-13 by IL-18 bispecific antibody for atopic dermatitis, into the clinic. The combined company will take the North Immunology name.

• Acquisition of Marea Therapeutics · Lisata Therapeutics

• Aethlon Medical & North Immunology Announce Merger · Aethlon Medical

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• Novartis halted autoimmune CAR-T trials after three patient deaths.

• The White House signed MFN pricing deals with nine more drugmakers.

• Lilly agreed to buy Merida Biosciences for up to $2.9bn.

• Ionis won the first FDA approval for Alexander disease.

• Ivonescimab beat Keytruda on overall survival in a China phase 3 trial.

Browse by day:

• Weekend catch-up: Wainua · Myqorzo · Mounjaro · Mimrylo · BioNTech

• Monday: Jazz Ziihera · Lilly-Merida · Repatha · US MFN · SEC-FDA · CAR-T deaths

• Tuesday: UK spinout · Vifor · MHRA · ARPA-H · GSK · Roche-Simcere · Remibrutinib

• Wednesday: Novartis-Alteogen · uniQure · FDA sites · Ultragenyx · Ivonescimab

• Thursday: GSK-HUTCHMED · AbbVie · eGenesis · Ionis

Weekend catch-up

Full data showed why AstraZeneca and Ionis’ Wainua failed in ATTR cardiomyopathy.

In CARDIO-TTRansform, presented at the European Society of Cardiology congress on Friday, Wainua (eplontersen) did not reduce cardiovascular death and recurrent cardiovascular events in 1,432 patients with transthyretin amyloid cardiomyopathy (rate ratio 0.89, p=0.277). The 57% already receiving a stabiliser showed no added primary-endpoint benefit, while patients not receiving one had fewer events.

• Eplontersen trial did not meet its primary endpoint in ATTR cardiomyopathy · ESC

Cytokinetics’ Myqorzo met both primary endpoints in non-obstructive hypertrophic cardiomyopathy.

In the phase 3 ACACIA-HCM trial, Myqorzo (aficamten) improved symptom scores and peak exercise capacity at week 36 against placebo. Cytokinetics plans a supplemental FDA filing in Q4 2026.

• Positive ACACIA-HCM results presented at ESC · Cytokinetics

The FDA approved Lilly’s Mounjaro to reduce cardiovascular risk in adults with type 2 diabetes.

The label covers heart attack, stroke and cardiovascular death in adults at high cardiovascular risk. In SURPASS-CVOT, Mounjaro (tirzepatide) was non-inferior to Trulicity (dulaglutide) for these events, with a hazard ratio of 0.92. Superiority was not established.

• FDA approves Mounjaro to reduce CV risk in adults with type 2 diabetes · Eli Lilly

The FDA approved Takeda’s Mimrylo, the first hepcidin mimetic for rare blood disorder polycythaemia vera.

In the phase 3 VERIFY trial, 76.9% of patients on Mimrylo (rusfertide) needed no phlebotomy over 32 weeks, against 32.9% on placebo.

• FDA approves first drug of its kind for polycythaemia vera · FDA

• FDA approves MIMRYLO for polycythaemia vera · Takeda

BioNTech and Genentech ended a phase 2 trial of their personalised colorectal cancer vaccine.

In resected stage II-III colorectal cancer positive for circulating tumour DNA, autogene cevumeran was being tested as monotherapy. An independent monitoring board found a numerical imbalance in overall survival and judged further continuation unlikely to change the efficacy outcome. It reported no new safety signal. A phase 2 trial in pancreatic cancer, pairing the vaccine with checkpoint inhibition and chemotherapy, continues. BioNTech shares fell 8.4%, wiping $2.6bn off its market value, reflecting concerns about personalised mRNA cancer vaccines as monotherapy.

• Update on phase 2 of autogene cevumeran in resected colorectal cancer · BioNTech

Monday 31 August

Jazz’s Ziihera produced further positive overall survival data in HER2-positive gastro-oesophageal cancer.

At the second interim analysis of phase 3 HERIZON-GEA-01, Ziihera (zanidatamab) plus chemotherapy showed a further improvement in overall survival versus Herceptin (trastuzumab) plus chemotherapy in first-line disease. The FDA had approved two Ziihera-based first-line regimens just six days earlier, on 25 August.

• Ziihera demonstrates significant OS benefit vs trastuzumab · Jazz Pharmaceuticals

• FDA approves Ziihera for HER2-positive gastro-oesophageal cancer · FDA

Lilly agreed to buy Merida Biosciences for up to $2.9bn.

Merida engineers biologics that selectively degrade the autoantibodies driving disease rather than suppressing the immune system. Its lead drug, MER511, is in phase 1 for Graves’ disease and thyroid eye disease. The all-cash price includes contingent milestones, and the deal is expected to close in Q4 2026.

• Lilly to acquire Merida Biosciences for autoimmune and allergic diseases · Eli Lilly

A prespecified analysis found Amgen’s Repatha cut mortality by 20% in high-risk patients without a prior heart attack or stroke.

In the phase 3 VESALIUS-CV trial in more than 12,000 patients, Repatha (evolocumab) added to lipid-lowering treatment reduced the risk of death by 20%. The benefit appeared after about 1.5 years.

• Repatha reduces risk of death in patients at risk of 1st heart attack/stroke · Amgen

Trump signed MFN pricing deals with nine more drugmakers.

The White House named Alcon, Astellas, BeOne Medicines, BridgeBio, CSL, Kyowa Kirin, Sun Pharma, Teva and UCB. Each agreed to give state Medicaid programmes most-favoured-nation prices and guarantee MFN prices on new medicines, taking the total to 26 companies, covering 89% of the branded US drug market. Incyte separately disclosed an agreement with the CMS under which Medicaid gets Jakafi (ruxolitinib) at prices aligned with selected international markets. Incyte expects to escape the proposed GUARD and GLOBE pricing models.

• Trump announces deals with nine additional pharmas · The White House

• Incyte Corporation Form 8-K, 31 August 2026 · SEC

The SEC and FDA agreed to share information on drug-related market disclosures.

Their memorandum of understanding sets protocols for exchanging information relevant to both agencies. SEC chairman Paul Atkins said FDA-related company disclosures “have a significant impact on our markets”.

• SEC & FDA announce MOU to bolster cooperation and ensure market integrity · SEC

Novartis halted autoimmune CAR-T trials after three patient deaths.

Novartis suspended trials of its CAR-T therapy rap-cel in lupus, myasthenia gravis, multiple sclerosis and other autoimmune conditions on 24 August after three cases of immune effector cell-associated haemophagocytic syndrome resulted in “fatal outcomes”. Its cancer trials continue. Bristol Myers Squibb also voluntarily paused enrolment in autoimmune trials of its CAR-T therapy zola-cel, “out of an abundance of caution”, after detecting “transient and reversible inflammatory events”. Both companies confirmed the suspensions directly to journalists on 31 August.

• Novartis, BMS pause autoimmune CAR-T trials on safety concerns · BioPharma Dive

• Novartis halts CAR-T trials after three deaths; BMS pauses studies · Fierce Biotech

Tuesday 1 September

UK ministers are considering an exit tax on university spinouts that move abroad, The Times reported.

The proposed levy would deter companies built with taxpayer money from taking intellectual property and jobs overseas. Minister Chris McDonald separately said British discoveries are developed abroad because start-ups cannot find enough investment or lab space at home.

• Ministers plot exit tax for university spinouts moving abroad · The Times

• UK science breakthroughs are being exploited overseas, minister says · The Times

The UK’s MHRA suspended Avacopan Vifor for new patients after doubts over its pivotal trial data.

The Medicines and Healthcare products Regulatory Agency found the benefit-risk balance of Avacopan Vifor, previously Tavneos, no longer positive after information raised concerns about the integrity and reliability of the study behind its approval. Existing patients get a six-month managed withdrawal, and the MHRA intends to revoke the licence on 1 March 2027.

• MHRA concludes Avacopan Vifor review reassessment of benefit-risk · GOV.UK

The UK government proposed powers for the MHRA to license medical devices directly.

One of three amendments tabled to the Health Bill would create the legal basis for a future regime under which devices sold in Great Britain through a domestic route could require an MHRA licence instead of third-party conformity assessment. Any regime would follow consultation, and UKCA marking remains in place in the meantime.

• MHRA regulatory reform amendments tabled in Government’s Health Bill · GOV.UK

ARPA-H committed up to $224m to AI for rare diseases and on-demand RNA medicine manufacturing.

The US agency announced awards under two programmes. RAPID will invest up to $98.5m over 4.5 years in AI tools and data resources for earlier rare disease diagnosis and drug development. GIVE will provide up to $125m to develop automated, distributed manufacturing systems capable of producing individualised RNA medicines on demand, with integrated quality control.

• Awards to advance AI for rare disease diagnosis, discovery and treatment · ARPA-H

• Teams selected to enable made-to-order genetic medicine manufacturing · ARPA-H

GSK plans to start a phase 3 trial of its mRNA flu vaccine this month.

Phase 2 data presented at the OPTIONS XIII flu conference showed higher immune responses against all tested strains than licensed standard- and high-dose vaccines. It will be the first phase 3 trial of an mRNA flu vaccine targeting both haemagglutinin and neuraminidase.

• mRNA seasonal flu vaccine advances to phase III after positive phase II data · GSK

Roche licensed Simcere Zaiming’s trispecific T-cell engager for $75m upfront and up to $1.5bn.

SIM0660 is a preclinical CD79a/CD19/CD3 T-cell engager designed for B-cell malignancies and autoimmune diseases. Roche gets exclusive global development, manufacturing and commercial rights, with Simcere also eligible for tiered royalties.

• Exclusive license agreement with Roche for SIM0660 global development · Simcere

Novartis’ remibrutinib beat teriflunomide in two phase 3 multiple sclerosis trials with no liver safety signal.

In REMODEL-1 and -2, the oral Bruton’s tyrosine kinase inhibitor reduced annualised relapse rates and brain lesions against teriflunomide in relapsing multiple sclerosis. Novartis plans regulatory submissions worldwide.

• Remibrutinib reduces relapse rates, & shows favourable safety in phase III · Novartis

Wednesday 2 September

Novartis signed an option and licence deal with Alteogen worth up to $3.2bn.

The agreement gives Novartis options to use Alteogen’s ALT-B4 enzyme to convert several intravenous biologics into subcutaneous injections. It is Alteogen’s fourth licensing deal this year, reinforcing the Korean company’s position as a competitor to Halozyme in subcutaneous drug delivery. The maximum value includes contingent option payments, milestones and royalties, with no upfront payment disclosed.

• Option & license agreement with Novartis for Hybrozyme SC products · Alteogen

uniQure filed its Huntington’s disease gene therapy with the FDA and the MHRA.

It seeks US accelerated approval of AMT-130 (ifezuntirgene inilparvovec) on three-year phase 1/2 data, and UK marketing authorisation. In March the FDA had queried whether the externally controlled data were sufficient for approval, before agreeing in July that submission under the accelerated approval pathway was reasonable.

• uniQure submits BLA for AMT-130 in Huntington’s disease · uniQure

FDA leaders set out tougher oversight of foreign clinical trial sites.

The heads of the agency’s drug, biologics, device and oncology centres wrote that the FDA can refuse data it cannot validate. It is adding resources for foreign inspections, becoming more systematic about telling sponsors and the public when sites are unavailable for inspection, and training reviewers to spot problem sites.

• Good Clinical Practices Are Not Optional · FDA

Ultragenyx shares fell 44% after its Angelman syndrome drug failed in phase 3.

The decline wiped approximately $1.15bn off the company’s market value, reducing it from $2.6bn to $1.5bn. In Aspire, apazunersen missed its primary endpoint on cognition and its key secondary endpoint. Ultragenyx will reassess the programme and plans “significant expense reductions”.

• Ultragenyx announces phase 3 Aspire results in Angelman syndrome · Ultragenyx

Ivonescimab beat Keytruda on overall survival in a China phase 3 trial.

Akeso and its partner Summit said a prespecified interim analysis of HARMONi-2 showed a statistically significant and clinically meaningful overall survival improvement for ivonescimab over Keytruda (pembrolizumab) in first-line PD-L1-positive non-small cell lung cancer. Detailed overall survival data were not disclosed and will be presented at a medical meeting. HARMONi-2 had previously met its progression-free survival primary endpoint. Shares in US partner Summit Therapeutics rose 17.3%, adding $2bn to take its market value to $13.7bn.

• Ivonescimab meets OS secondary endpoint in phase III HARMONi-2 · Akeso

• Ivonescimab shows OS benefit vs Keytruda in HARMONi-2 · Summit Therapeutics

Thursday 3 September

GSK licensed HUTCHMED’s KRAS-EGFR conjugate for $110m upfront.

The deal for HMPL-A830, worth up to $1.3bn, covers the world excluding China, Hong Kong, Macau and Taiwan. The drug delivers a KRAS inhibitor payload through an anti-EGFR antibody, with clinical development expected to begin in H2 2026.

• Licensing agreement with GSK for KRAS-EGFR Ab conjugate therapy · HUTCHMED

AbbVie’s etentamig met both primary endpoints in phase 3 multiple myeloma.

At the first planned interim analysis of CERVINO in triple-class exposed relapsed or refractory myeloma, the BCMAxCD3 bispecific produced a 74% response rate against 45.7% with standard therapies and cut the risk of progression or death by 60%.

• Positive topline results from phase 3 CERVINO trial of etentamig · AbbVie

Three patients have lived more than eight months without dialysis on eGenesis engineered pig kidneys.

Five patients at Mass General Brigham have received EGEN-2784 since March 2024. Two later moved to human donor kidneys, the first such transitions reported, and the phase 1/2/3 RESTORE trial is due to start in Q1 2027.

• Clinical update from study of EGEN-2784 in patients with kidney failure · eGenesis

The FDA approved Ionis’ Zanvastro, the first drug for Alexander disease.

Zanvastro (zilganersen) is an antisense drug injected into the spinal canal every three months for children and adults with the rare, progressive neurological disorder. In a randomised study of 49 patients, treated patients aged five and over with measurable walking difficulty at baseline walked faster at 61 weeks than untreated ones.

• FDA approves first drug to treat Alexander disease · FDA

• Zanvastro FDA approved as 1st disease-modifying drug for Alexander disease · Ionis

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

• FDA approves RevMed pancreatic cancer drug, the first that targets RAS.

• Akeso’s ivonescimab beats Imfinzi on biliary cancer survival.

• FDA approves Jazz’s Ziihera for first-line stomach cancer.

• Roche and Hanmi agree $2.3bn obesity deal.

• PhRMA loses Medicare price negotiation appeal.

Browse by day:

• Weekend: China trials · Vaccines · Guardant

• Monday: Roche-Hanmi · GSK · Roche · Regenxbio · Capricor · J&J

• Tuesday: Akeso · Ziihera · McKesson · Abbott · Antibodies

• Wednesday: RevMed · Biohaven · Medicare · Shingrix

• Thursday: Tezspire · Lisraya · Gilead

• Friday: HUTCHMED Atled

Weekend catch-up

Two Republican lawmakers asked the FDA to reject clinical data from Chinese sites that it had not recently inspected.

House China committee chairman John Moolenaar and Congressman Ben Cline asked the FDA to accept data from Chinese trial sites only if it had conducted an in-person inspection within the previous 12 months. They also asked for a published assessment of how far US approvals rely on Chinese investigator-initiated trials, following reports of three patient deaths in such trials.

• Chinese clinical data risks · Select Committee on the CCP

HHS opened a consultation, running to 20 September, on how US childhood vaccine recommendations are categorised.

The request for information, signed by HHS Secretary Robert F. Kennedy Jr., asks whether the existing recommendation categories should be changed or supplemented. It follows a Trump executive order of 10 August on childhood vaccine recommendations.

• Categories in federal vaccine recommendations · Federal Register

A Delaware federal court ordered Guardant Health to pay TwinStrand and the University of Washington more than $245m over sequencing patents.

The final judgment preserved a 2023 jury verdict that Guardant wilfully infringed two patents underlying TwinStrand’s Duplex Sequencing technology. It awarded more than $245.2m in damages, accrued royalties and interest and set a 6% royalty on covered sales until the patents expire in March 2033. Guardant said on Monday that it would appeal, with execution of the judgment and collection of royalties stayed pending appeal.

• $245m patent judgment against Guardant · TwinStrand

• Guardant to appeal patent judgment · Guardant

Monday 24 August

Genentech agreed to pay up to $2.3bn for a Hanmi obesity candidate designed to preserve lean mass.

Genentech will pay $190m upfront for worldwide rights outside South Korea to HM17321, a urocortin-2 analogue designed to reduce weight while preserving lean mass. Hanmi will complete the phase 1 trial before Genentech takes over development from phase 2.

• Genentech licenses obesity drug · Hanmi

GSK received the first approval for its hepatitis B drug Hibsago, in Japan.

Japan’s health ministry cleared Hibsago (bepirovirsen), an antisense drug licensed from Ionis, as a functional cure for chronic hepatitis B in adults who have taken nucleos(t)ide analogue antivirals for at least six months and start treatment with HBsAg ≤3,000 IU/mL and HBV DNA <90 IU/mL. In the B-Well phase 3 trials 19% of patients with HBsAg ≤3,000 IU/mL reached functional cure. Decisions elsewhere, including the US, are due in the coming months.

• Japan approves hepatitis B drug Hibsago · GSK

The FDA cleared Roche’s Alzheimer’s blood test Elecsys pTau217, developed with Lilly.

It is the first FDA-cleared single-biomarker blood test that can both rule in and rule out amyloid pathology, in primary as well as specialty care, for people aged 55 and over with cognitive decline. It is designed to run across Roche’s installed base of more than 4,500 cobas analysers in US labs.

• FDA clears Alzheimer’s blood test · Roche

The FDA put Regenxbio’s Hunter syndrome gene therapy on clinical hold.

Spine scans found small nodules or cystic masses in five boys treated with RGX-121 three to six years ago, which investigators judged non-serious and likely benign. Regenxbio no longer expects to refile soon and is concentrating on Duchenne, where it plans to file this quarter.

• RGX-121 regulatory update · REGENXBIO

The FDA gave Capricor’s Duchenne cell therapy three more months of review.

The decision on deramiocel moves to 22 November. After a July advisory committee meeting, Capricor filed 24-month open-label extension data from the phase 3 HOPE-3 trial to support a refined proposed indication focused on upper limb function. The FDA classed the submission as a major amendment.

• Deramiocel review extended · Capricor

J&J’s Imaavy became the first approved treatment for warm autoimmune haemolytic anaemia.

The FDA cleared Imaavy (nipocalimab), already approved in myasthenia gravis, for patients aged 12 and over currently or previously treated with corticosteroids. In the phase 2/3 ENERGY study about three times as many patients on the approved dose achieved a durable haemoglobin response as on placebo.

• FDA approves Imaavy for wAIHA · J&J

Tuesday 25 August

Akeso reported an overall survival benefit for ivonescimab over Imfinzi in biliary tract cancer.

In the China-only phase 3 HARMONi-GI1 trial, the PD-1/VEGF bispecific plus chemotherapy showed a statistically significant overall survival benefit over Imfinzi (durvalumab) plus chemotherapy at an interim analysis in first-line advanced biliary tract cancer. It also met its progression-free survival and response-rate goals.

It is ivonescimab’s first phase 3 readout outside lung cancer. Partner Summit, which holds rights in territories including the US and Europe, noted that Akeso alone generated and analysed the data. Full results are due at a medical congress.

• Ivonescimab survival benefit in biliary cancer · Summit

The FDA approved Jazz’s Ziihera in first-line HER2+ stomach cancer.

The FDA cleared Ziihera (zanidatamab), Jazz Pharmaceuticals’ HER2-targeted bispecific antibody, for first-line treatment of HER2-positive gastric, gastrooesophageal junction and oesophageal adenocarcinoma in two regimens: with chemotherapy and BeOne’s Tevimbra (tislelizumab) for IHC 3+ or IHC 2+/ISH+ disease, and with chemotherapy alone for IHC 3+ disease only. In the HERIZON-GEA-01 trial the three-drug regimen extended median overall survival to 26.4 months from 19.2 months on Herceptin and chemotherapy. Two Roche companion diagnostics were approved alongside it.

• FDA approves first-line Ziihera · FDA

• FDA approves first-line Ziihera · Jazz Pharmaceuticals

McKesson agreed to buy Precision Medicine Group for $2.25bn.

Clinical research and commercialisation services provider Precision Medicine Group will report within McKesson’s Oncology & Multispecialty segment once the deal closes, subject to regulatory clearance.

• Acquisition of Precision Medicine Group · McKesson

Abbott received FDA authorisation for the first sensor that tracks both glucose and ketones.

Libre Duo 10 Day warns people with diabetes of rising ketones that can lead to diabetic ketoacidosis, alongside continuous glucose readings. It is cleared for ages 2 and over, and Abbott plans a US launch later this year.

• FDA authorises dual glucose-ketone sensor · Abbott

An audit documented nearly 19,000 apparently manipulated or otherwise problematic validation images across 15 antibody suppliers.

In an audit published on 25 August, Reese Richardson documented 18,943 images across 17,495 antibody products. The examples include repeated background noise, painted-over Western blots and images reused across products or brands. Richardson cautioned that the work was not a complete audit of any supplier’s catalogue and that original high-resolution images would be needed to establish falsification.

• 15 antibody companies use faked antibody validation data · Reese Richardson

Wednesday 26 August

The FDA approved Revolution Medicines’ pancreatic cancer drug Rasonque.

Rasonque (daraxonrasib), a RAS inhibitor, is cleared for adults with metastatic pancreatic adenocarcinoma who have had at least one prior systemic therapy or cannot take multi-drug chemotherapy. The once-daily pill is on sale now at a wholesale acquisition cost of $39,800 for 30 days.

The approval came 6.5 months before the PDUFA date under the Commissioner’s National Priority Voucher pilot and rests on the phase 3 RASolute 302 trial against chemotherapy. Median overall survival was 13.2 months on Rasonque against 6.7 months on standard chemotherapy. The FDA called it a first-in-class targeted therapy for metastatic pancreatic cancer.

• FDA approves Rasonque · Revolution Medicines

• First RAS-targeted pancreatic cancer therapy · FDA

Biohaven licensed its Kv7 epilepsy platform, led by opakalim, to SK Biopharmaceuticals for a $400m upfront fee.

SK pays $350m at closing and $50m a year later, plus up to $150m in milestones and royalties of up to the low twenties per cent on US sales. It also takes on up to $245m in milestones owed to Knopp Biosciences. Biohaven keeps running the phase 2/3 trials and will file for approval.

• Biohaven-SK epilepsy licensing deal · Biohaven

A federal appeals court rejected PhRMA’s constitutional challenge to Medicare drug price negotiation.

The Fifth Circuit upheld a district court ruling against the suit PhRMA brought with the National Infusion Center Association and the Global Colon Cancer Association. Judge Leslie Southwick, agreeing with the Second Circuit, wrote that manufacturers “lack a protected interest in selling to Medicare beneficiaries at a preferred price” because taking part in Medicare is voluntary.

On Monday a federal judge in Washington had also ruled against Merck, rejecting its arguments that the programme breaches the First and Fifth Amendments.

• National Infusion Center Association v. Kennedy · Fifth Circuit

• Merck v. Kennedy · US District Court

An Oxford study linked Shingrix to a lower cardiovascular disease burden than Zostavax.

Comparing more than 70,000 US adults vaccinated either side of the 2017 switch from Zostavax to Shingrix, researchers found Shingrix recipients had a 9% lower cardiovascular disease burden over seven years, including 10% less coronary heart disease and 12% less heart failure. A lower stroke risk was seen in men. The researchers said clinical trials are needed before shingles vaccination could be recommended for preventing cardiovascular disease.

• Shingrix and cardiovascular risk · Nature Medicine

• Shingrix linked to lower heart disease risk · University of Oxford

Thursday 27 August

Tezspire met both co-primary and all key secondary endpoints in a phase 3 eosinophilic oesophagitis trial.

In CROSSING, AstraZeneca and Amgen’s Tezspire (tezepelumab) showed statistically significant and clinically meaningful improvements versus placebo at week 24 in histologic remission and in the frequency and severity of difficulty swallowing, with improvements across all key secondary endpoints. The effects were sustained to week 52 at both doses tested.

• Tezspire phase 3 EoE results · AstraZeneca

The FDA approved Roivant’s Priovant drug Lisraya, the first targeted treatment for dermatomyositis.

Lisraya (brepocitinib), a first-in-class dual TYK2/JAK1 inhibitor, is approved for adults with dermatomyositis. In the phase 3 VALOR trial 55% of patients on Lisraya reached at least moderate improvement on the Total Improvement Score with minimal or no steroid use, against 30% on placebo.

• FDA approves Lisraya · Roivant

The FDA approved Gilead’s Bixlenvo, a once-daily HIV pill pairing bictegravir with lenacapavir.

Bixlenvo is cleared as a complete regimen for virologically suppressed adults on stable antiretroviral therapy with no known or suspected resistance to either component, including people on complex regimens who cannot take existing single-tablet options. Treatment starts with two days of Bixlenvo plus oral Sunlenca, followed by Bixlenvo alone once daily.

• FDA approves Bixlenvo · Gilead

Friday 28 August

China conditionally approved HUTCHMED’s Atled for FGFR2-altered intrahepatic cholangiocarcinoma.

Atled (fanregratinib), an oral FGFR1-3 inhibitor, is cleared for previously treated adults with advanced, metastatic or unresectable intrahepatic cholangiocarcinoma with FGFR2 fusions or rearrangements. In the phase 2 registration cohort 42.5% of patients responded and median overall survival was 16.6 months.

• Approval for Atled · HUTCHMED

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• Moderna’s mRNA delivered landmark phase 3 cancer data, adding $50bn in value.

• Trump nominated White House aide Heidi Overton to run the FDA.

• AstraZeneca won two late-stage lung cancer trials and stopped another.

• argenx’s Vyvgart Hytrulo met its phase 3 myositis endpoint.

• Regeneron won FDA approval for FOP drug Pasatru.

Browse by day:

• Weekend catch-up: Skye-Redx · Lantheus Alzheimer’s · Biogen felzartamab

• Monday: AZ lung trials · argenx · EyePoint · Sandoz-Henlius · Slate-Fulcrum

• Tuesday: Amylyx · LEO-Tanabe · BioMarin-Alesta · Teva · Biokin · China trial deaths

• Wednesday: Moderna-Merck · Regeneron · Ultragenyx · US FDA pick · Aurinia-Teva

• Thursday: UK weekly insulin

Weekend catch-up

Redx Pharma agreed a reverse merger with Nasdaq-listed Skye Bioscience to create Fibrx Therapeutics, backed by $125m of concurrent financing.

The deal finally gives Redx the US public-market listing it has pursued since its failed 2023 reverse with Jounce and 2024 AIM delisting. Fibrx will be built around Redx's fibrosis pipeline and led by its management. The $125m is expected to fund it into 2029 and through phase 2 topline data for lead asset RXC008 in H2 2028.

• Skye Bioscience & Redx Pharma transaction and $125m financings · Skye Bioscience

The FDA approved Lantheus’ tau PET imaging agent for Alzheimer’s disease.

Tauklarify (florquinitau F 18, previously MK-6240) is cleared to image tau neurofibrillary tangle pathology in adults with cognitive impairment being evaluated for Alzheimer’s disease, complementing amyloid PET. The approval comes as Lantheus is being acquired by radiopharma rival Curium in a deal worth up to $8bn.

• FDA approval of TAUKLARIFY Tau PET Imaging Agent for Alzheimer’s · Lantheus

Biogen’s antibody felzartamab won its first approval anywhere, in China.

The anti-CD38 antibody was approved for multiple myeloma, its first authorisation anywhere. More important for Biogen, felzartamab is also in global phase 3 trials in immune-mediated diseases, where the company sees the larger opportunity.

• TJ Biopharma & Biogen first global approval of felzartamab China · TJ Biopharma

Monday 17 August

AstraZeneca won two late-stage lung cancer trials and stopped another.

In DESTINY-Lung04, Enhertu, partnered with Daiichi Sankyo, improved progression-free survival (PFS) over chemotherapy plus Keytruda as first-line treatment of HER2-mutant advanced non-small cell lung cancer. In SAFFRON, Tagrisso plus Orpathys, partnered with HUTCHMED, improved PFS and overall survival in EGFR-mutated disease with high MET overexpression after progression on Tagrisso.

The data extend AstraZeneca’s position in molecularly defined lung cancer, although they came on the same day it discontinued a separate Phase 3 trial of volrustomig.

• Enhertu improved survival 1st-line advanced NSCLC DESTINY-Lung04 · AstraZeneca

• Tagrisso+Orpathys improved survival in EGFR-mutated lung cancer · AstraZeneca

• Update on eVOLVE-Lung02 Phase III of volrustomig in NSCLC · AstraZeneca

argenx’s Vyvgart Hytrulo met its phase 3 endpoint in myositis.

In the ALKIVIA trial, Vyvgart Hytrulo (efgartigimod) delivered a 15.4-point greater improvement in mean Total Improvement Score at week 52 than placebo (p=0.0011) across the combined immune-mediated necrotising myopathy and dermatomyositis population, with benefit from week 4. It is the first phase 3 trial to show a benefit in immune-mediated necrotising myopathy, which has no approved therapy.

• Positive Topline Results Phase 3 ALKIVIA efgartigimod autoimmune myositis · argenx

EyePoint’s Duravyu missed its primary endpoint in wet AMD.

In LUGANO, the first of two phase 3 trials, Duravyu (vorolanib) failed to meet its prespecified non-inferiority endpoint against Eylea on mean change in best corrected visual acuity. EyePoint said the result was affected by nine patients, about 4% of the cohort, with vision loss it considered unrelated to wet AMD. An ad hoc analysis excluding those patients met the non-inferiority threshold. The company still plans to file in H1 2027, subject to results from the identical LUCIA trial due in Q4 2026.

• Topline Data LUGANO, first of two Phase 3 trials DURAVYU Wet AMD · EyePoint

Sandoz agreed to pay up to $322m for three Henlius biosimilars.

Shanghai Henlius granted Sandoz exclusive registration and commercialisation rights outside China to as many as 10 proposed antibody and antibody-drug conjugate biosimilars, with proposed cetuximab, evolocumab and belimumab copies first. Sandoz pays up to $322m in total, of which up to $100.5m is invoiced in 2026.

• Sandoz strategic collaboration unlock value of biosimilars Platform · Henlius

Slate agreed a merger with Fulcrum Therapeutics and $245m in commitments.

The all-stock merger would take migraine developer Slate onto Nasdaq, alongside a committed $245m private placement led by Frazier Life Sciences. The combined company will retain the Slate name. Lead asset SLTE-1009 is a subcutaneous anti-PACAP/VIP antibody, and the financing is expected to fund operations into 2029.

• Fulcrum Therapeutics and Slate Medicines announce merger agreement · Fulcrum

Tuesday 18 August

Amylyx’s GLP-1 antagonist avexitide cut hypoglycaemic events by more than half in phase 3.

In the LUCIDITY trial in 78 patients with post-bariatric hypoglycaemia after gastric bypass, avexitide reduced the composite rate of Level 2 and Level 3 hypoglycaemic events by 55% versus placebo (p=0.000003) and met every secondary endpoint. Amylyx plans to file by year-end for a condition with no approved treatment.

• Positive topline Phase 3 LUCIDITY Avexitide Post-Bariatric Hypoglycemia · Amylyx

LEO Pharma agreed to acquire worldwide rights to dersimelagon from Tanabe Pharma for up to $435m.

Dersimelagon is an oral MC1R agonist for erythropoietic protoporphyria and X-linked protoporphyria. It has completed phase 3, is filed and awaits an FDA decision. The deal gives LEO a late-stage rare-disease asset close to potential launch in dermatology.

• LEO Pharma strengthens late-stage pipeline acquisition dersimelagon · LEO Pharma

BioMarin agreed to buy Alesta Therapeutics for $275m upfront.

The deal brings ALE1, an oral small molecule that could become the first oral therapy for hypophosphatasia, a rare bone disease. Alesta will spin out its other assets before closing, expected this quarter. ALE1 adds to BioMarin’s skeletal conditions portfolio.

• Acquiring Alesta Therapeutics ALE1, potential first oral Hypophosphatasia · BioMarin

A federal appeals court handed Teva a partial win against Medicare price negotiations.

The US Court of Appeals for the D.C. Circuit sent back to a lower court the question of whether CMS's “bona fide marketing” standard, which determines when a drug becomes eligible for negotiation, complies with the Inflation Reduction Act. The court did not rule that the standard itself was unlawful. Teva's broader constitutional challenges were unsuccessful.

• Teva Pharmaceuticals v. Kennedy, No. 25-5425, opinion · US Court of Appeals

• Teva scores rare partial win in CMS price negotiation appeal · Fierce Pharma

Biokin’s iza-bren, partnered with BMS, met its phase 3 endpoint in lung cancer.

In the China BL-B01D1-301 trial, the EGFR×HER3 bispecific ADC met its primary progression-free survival endpoint at an interim analysis in EGFR-mutated non-small cell lung cancer after EGFR-TKI treatment, with a positive trend in overall survival. It is the fourth phase 3 study in which iza-bren has met its primary endpoint.

• Meeting primary endpoint interim analysis phase III iza-bren NSCLC · SINA

• Biokin, BMS’ bispecific ADC hits goal in China lung cancer trial · Fierce Pharma

A third patient death was reported in China's investigator-initiated cell and gene therapy trials.

Endpoints reported a previously undisclosed death in a CAR-T study, following two recently reported deaths in experimental gene-therapy trials. The cases have increased scrutiny of transparency and oversight in China’s investigator-initiated trial (IIT) system.

• Third death revealed in China’s popular but opaque trials · Endpoints News

• After 2 deaths, can China’s alternative regulatory path be trusted? · Fierce Biotech

Wednesday 19 August

Moderna’s mRNA cancer therapy met two phase 3 endpoints in melanoma.

In INTerpath-001, intismeran autogene, Moderna’s personalised mRNA neoantigen therapy, plus Merck’s Keytruda beat Keytruda alone on recurrence-free survival and distant metastasis-free survival at a prespecified interim analysis in patients with completely resected stage IIB-IV melanoma. The trial continues to evaluate other secondary endpoints, including overall survival.

It is the first positive phase 3 trial of an individualised neoantigen therapy and the first for an mRNA cancer therapy. The result validates a personalised treatment model in which tumour sequencing, computational neoantigen selection and patient-specific mRNA manufacturing are combined to generate anti-tumour T-cell responses in vivo. Moderna shares rose 177%, adding about $45bn to its market value, reflecting the broader potential of the platform beyond melanoma.

• Merck and Moderna Announce Phase 3 INTerpath-001 Trial of Intismeran Autogene Plus KEYTRUDA Met Endpoints of RFS and DMFS in Melanoma · Moderna

Regeneron won FDA approval for FOP drug Pasatru.

Pasatru, an antibody targeting Activin A, was approved to reduce new heterotopic ossification lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva, the ultra-rare disease in which soft tissue turns to bone. In the phase 3 trial, two doses reduced new lesions by 90% and 94% vs placebo at 56 weeks.

• Pasatru first only FDA-approved heterotopic ossification lesions FOP · Regeneron

Ultragenyx won its first gene therapy approval, in a rare metabolic disease.

The FDA granted accelerated approval to Genglycos for patients eight and older with glycogen storage disease type Ia. Genglycos is designed to address enzyme deficiency and reduce dependence on cornstarch used to maintain blood glucose. It is Ultragenyx’s first marketed gene therapy, and came with a Priority Review Voucher.

• Ultragenyx announces U.S. FDA approval of GENGLYCOS gene therapy · Ultragenyx

Trump picked Heidi Overton to run the FDA.

The president nominated White House policy aide Heidi Overton, M.D., Ph.D., as FDA commissioner. She faces Senate confirmation and would take over an agency that has lost around one in six of its staff over the past year (>3,000), after months of leadership turnover and regulatory controversy. Reuters reported concern among drugmakers and FDA staff over her limited management experience.

• Trump selects Heidi Overton as FDA commissioner · Truth Social

• Trump picks White House ally Overton to lead embattled FDA · Reuters

The FDA cleared the first standalone robotic blood-draw device.

The FDA authorised Vitestro's Aletta through the De Novo pathway, the first standalone robotic device able to draw blood from a patient's arm without hands-on intervention. It uses near-infrared imaging and ultrasound to locate a vein and automates the draw. A phlebotomist supervises up to three devices, with sensors that stop or pause the procedure if the patient moves or another safety issue is detected. FDA said success rates were comparable with or better than trained phlebotomists.

• FDA Authorizes First-of-Its-Kind Robotic Blood Draw Device · FDA

A US appeals court sided with Parse Biosciences over 10x Genomics on patents.

The Federal Circuit upheld the Patent Trial and Appeal Board's invalidation of three 10x Genomics patents in its dispute with Qiagen-owned Parse Biosciences. The ruling removes three patents from 10x's enforcement arsenal in the single-cell sequencing market, where the two companies compete directly.

• 25-1199: 10X Genomics, Inc. v. Parse Biosciences, Inc. [Opinion], Nonprecedential · US Court of Appeals for the Federal Circuit

Aurinia settled with Teva over Lupkynis generic entry in December 2036.

The settlement allows Teva to launch its generic version of Lupkynis (voclosporin) in the US no earlier than 7 December 2036, subject to defined contingencies and FDA approval. Teva also acknowledged two Aurinia patents as valid, enforceable and infringed by its proposed product. Aurinia continues to litigate separate generic challenges from other companies, so the agreement does not resolve all generic risk.

• Aurinia Settles Patent Litigation with Teva · Aurinia Pharmaceuticals

• Aurinia deal with Teva keeps Lupkynis generic in check until ‘36 · Fierce Pharma

Thursday 20 August

NICE backed Lilly’s weekly insulin Onswik for the NHS.

The cost watchdog recommended insulin efsitora alfa, also known as Onswik, for type 2 diabetes in final draft guidance. It controls blood glucose as well as widely used long-acting insulins while cutting injections by 85%, from daily to once a week. NICE said it could particularly benefit people who rely on a carer or nurse for injections. The recommendation remains subject to MHRA licensing before NHS use.

• Weekly insulin jab could cut injections from 365 a year to just 52 · NICE

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• The White House ordered an overhaul of US childhood vaccination schedules.

• The FDA proposed 50% fee cuts for US-run trials.

• Jazz and Actio agreed a $1.3bn rare-epilepsy deal.

• Sionna dropped SION-719 after a phase 2 miss.

• Definium’s LSD pill cleared a second phase 3.

Browse by day:

• Weekend catch-up: WuXi ruling

• Monday: US vaccines · Jazz-Actio · Sionna · UK Foundayo · MoonLake · Sobi-Innate

• Tuesday: Autolus · Vaderis · Vykat XR

• Wednesday: Definium · Cullinan-Taiho · MMR pushback · Sangamo auction

• Thursday: US user fees · Tavneos

Weekend catch-up

A US federal judge blocked the Pentagon from enforcing WuXi AppTec’s designation as a Chinese military company.

A US District Court granted a preliminary injunction barring the Department of Defense from effecting the Section 1260H listing while WuXi’s challenge runs, finding the company likely to show the designation was arbitrary and capricious. WuXi had documented clients cancelling and moving work to rivals since the June listing.

• WuXi AppTec Co., Ltd. v. US Department of Defense, 1:26-cv-02069 · CourtListener

• Judge blocks listing of WuXi AppTec as ‘Chinese military’ · Pharma Manufacturing

Monday 10 August

The White House ordered a rewrite of US childhood vaccine schedules.

The executive order, “Delivering Gold Standard Childhood Vaccine Recommendations for Americans”, cuts the universally recommended childhood vaccinations from 17 to 11, moves shots including hepatitis B, rotavirus, influenza and COVID-19 to high-risk or shared decision-making categories, and directs that the MMR vaccine be given as three separate shots once single-disease versions exist in the US.

• Gold standard childhood vaccine recommendations for Americans · White House

Jazz agreed to buy Actio Biosciences for up to $1.32bn.

Jazz Pharmaceuticals will pay $820m upfront and up to $500m in contingent consideration for the private biotech, whose lead asset ABS-1230 is a potential first-in-class KCNT1 ion channel inhibitor for a rare genetic epilepsy affecting 2,500 in the US. The deal extends the epilepsy business built on Epidiolex (cannabidiol, GW Pharma).

• Acquiring Actio Biosciences, expanding rare epilepsy portfolio · Jazz Pharmaceuticals

Sionna’s cystic fibrosis add-on failed.

In the phase 2a PreciSION CF trial, SION-719 added to Trikafta shifted sweat chloride by a placebo-adjusted -1.0 mmol/L (p=0.7). Sionna Therapeutics will not take the NBD1 stabiliser forward as an add-on to standard of care. Sionna named SION-451 plus SION-2222 as its preferred pairing while it weighs next steps.

• Topline data from two development programs in CF · Sionna Therapeutics

The MHRA authorised Lilly’s orforglipron, the first regulator in Europe to clear it.

Foundayo (orforglipron) is authorised for weight loss in adults with obesity or overweight plus a comorbidity, and separately for inadequately controlled type 2 diabetes. It is the second oral GLP-1 cleared for weight loss in the UK in two months, after Novo Nordisk’s oral Wegovy in June. NHS use will require NICE evaluation first.

• UK first in Europe to authorise orforglipron for weight loss and diabetes · MHRA

MoonLake’s sonelokimab met its endpoints in first phase 3 in psoriatic arthritis.

In IZAR-1, 42.1% of biologic-naive patients on the nanobody’s 60 mg induction regimen reached ACR50, with wins across key secondary endpoints and no new safety signals. The trial runs to week 52, reading out fully in H1 2027; IZAR-2, in TNF-inadequate responders with a Skyrizi reference arm, completes enrolment in Q3 2026.

• Positive topline results phase 3 of sonelokimab · MoonLake Immunotherapeutics

Sobi licensed Innate Pharma’s lacutamab in a deal worth up to $580m.

Sobi pays $75m at closing for global rights to commercialise the anti-KIR3DL2 antibody on any accelerated approval in Sézary syndrome, with up to $40m in near-term milestones and up to $465m tied to an option over full development rights.

• Partnership with Innate Pharma to license lacutamab in T-cell lymphoma · Sobi

The FDA rejected ITM’s radiopharmaceutical over manufacturing issues.

The complete response letter for 177Lu-edotreotide (ITM-11) in gastroenteropancreatic neuroendocrine tumours rests on chemistry, manufacturing and controls (CMC) deficiencies and unresolved conditions at a third-party facility. It raised no clinical or safety concerns and asks for no new trials. The phase 3 COMPETE trial had beaten everolimus on progression-free survival. ITM intends to resubmit.

• ITM Receives Complete Response Letter for 177Lu-edotreotide (ITM-11) · ITM

Revolution Medicines and BeOne Medicines agreed a RAS collaboration.

BeOne takes exclusive rights to Revolution’s four clinical RAS(ON) inhibitors in selected Asian markets ex-Japan and South Korea and will fund a global registrational phase 3. The pair will also test combinations pairing daraxonrasib and zoldonrasib with BeOne’s PRMT5 inhibitor and its EGFR x MET x MET trispecific antibody.

• BeOne clinical and regional commercialization collaboration · Revolution Medicines

Silence Therapeutics’ divesiran hit in phase 2 and it priced a $175m offering.

In the 48-patient SANRECO polycythaemia vera trial, 88% on the siRNA responded against 19% on placebo, while phlebotomies fell to 0.2 against 2.1 (per pt, p<0.0001). Phase 3 starts H1’27. Silence priced an upsized $175m offering at $13.50 the next day.

• Positive topline results for divesiran & $175m offering · Silence · Silence

AbCellera’s mAb cut hot flushes 83% in phase 2 and it priced a $200m offering.

A single 600 mg dose of ABCL635 cut daily moderate-to-severe hot flushes by 83% reduction against 33% placebo, with no serious adverse events among 92 postmenopausal women. AbCellera priced an oversubscribed $200m offering at $9.75.

• Positive topline results for ABCL635 & $200m offering · AbCellera · AbCellera

Quanterix cut its 2026 revenue guidance by 15% and its shares fell about 40%.

Q2 revenue of $32.9m came in 9% below the company’s own May guidance, with pro forma revenue down 23% YoY, and the full-year range falls to $142m-$148m from $169m-$174m on weak academic and government demand and execution challenges.

• Quanterix releases financial results for the second quarter of 2026 · Quanterix

Tuesday 11 August

Autolus confirmed Q2 Aucatzyl revenue of $45.7m, up 119% YoY.

Q2 net revenue for Aucatzyl reached $45.7m, up 119% YoY, taking H1 to $71.9m; the London-based company guides 2026 sales of $140-$150m. Enrolment continues in lupus nephritis, paediatric acute lymphoblastic leukaemia and multiple sclerosis.

• Second quarter 2026 financial results and business updates · Autolus Therapeutics

Vaderis raised a $152m series B and started a phase 3 in a rare vascular disorder.

The Swiss biotech’s engasertib, an oral allosteric AKT inhibitor, enters the global phase 3 HEROIC study in hereditary haemorrhagic telangiectasia, which has no approved treatment anywhere. Goldman Sachs Alternatives and TCGX co-led the round.

• Oversubscribed $152m Series B financing & Phase 3 study · Vaderis Therapeutics

Prader-Willi organisations flagged serious adverse events reported on Neurocrine’s Vykat XR.

A joint statement from PWSA USA, the Foundation for Prader-Willi Research and IPWSO urges careful patient selection and monitoring after post-marketing reports of oedema, respiratory and cardiac complications. Fierce Pharma reported seven deaths and >100 serious adverse events in the FDA’s adverse event system. Neurocrine, which took on Vykat XR (diazoxide choline) in its $2.9bn Soleno purchase, says the drug has “a compelling risk-benefit profile in the context of a very serious disease.”

• PWS organizations share joint statement on VYKAT XR adverse events · PWSA USA

• Neurocrine stands by blockbuster hopeful as docs raise safety flags · Fierce Pharma

Wednesday 12 August

Definium’s LSD pill cleared its second phase 3.

In the Voyage trial in generalised anxiety disorder, DT120 (lysergide) 100 µg cut the Hamilton Anxiety score by 11.6 points against 6.2 on placebo at week 12 (p<0.0001). It is the second positive phase 3 for the orally disintegrating tablet after June’s Emerge readout in major depression. The FDA has granted breakthrough therapy designation in anxiety. A second trial, Panorama, reads out in September.

• Topline results from Phase 3 Voyage study of DT120 ODT · Definium Therapeutics

Taiho and Cullinan’s zipalertinib met its phase 3 goal in first-line lung cancer.

In a planned interim analysis of the 285-patient REZILIENT3 trial, the EGFR inhibitor plus chemotherapy improved progression-free survival in untreated EGFR exon 20 insertion non-small cell lung cancer. Pending FDA discussions, the partners plan to pursue US approval, which would take the drug into Rybrevant’s (J&J) territory.

• Zipalertinib meets primary endpoint of PFS in Phase 3 REZILIENT3 · Taiho Oncology

Merck and GSK pushed back on splitting the MMR vaccine.

Both makers of the US combination shot told Reuters there is no published evidence to support separating it, and neither set out plans to. Merck said single-disease approvals could take “as many as 10“ years. No standalone measles, mumps or rubella shot has been sold in the US since Merck stopped making them in 2008.

• Goal to split MMR vaccine could take a decade, experts say · Reuters via KFGO

Sangamo selected PTC and Lilly as winning bankruptcy-auction bidders.

PTC bid $111m at closing plus up to $100m in approval milestones for the Fabry disease gene therapy isaralgagene civaparvovec. Lilly takes the capsid, zinc finger and MINT platforms and the prion disease programme for $50m. Winning bids total $163.6m in cash, subject to approval by the US Bankruptcy Court (Delaware) in Q3.

• Sangamo selects successful bidders following asset auction · Sangamo Therapeutics

Thursday 13 August

The FDA proposed a 50% fee cut for US-based phase 1 trials.

The proposed PDUFA VIII commitment letter, covering fiscal years 2028 to 2032, would halve the application fee where a filing includes clinical data from at least one phase 1 trial run in the US and begun after 1 October 2027.

• PDUFA VIII: Fiscal Years 2028-2032 · FDA

EMA says Tavneos approval relied on “incorrect and misleading” trial data.

The CHMP recommended revoking Tavneos (avacopan) in June, with the European Commission making that decision legally binding across the EU on 4 August. The EMA has now published the grounds for that decision, concluding that the ADVOCATE trial supporting approval breached good clinical practice and that its data could no longer be relied on to demonstrate effectiveness. CSL’s Vifor unit is managing the withdrawal.

• Tavneos: Article 20 referral · European Medicines Agency

• EC adopts decision to revoke marketing authorisation for TAVNEOS (avacopan) · CSL

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• AstraZeneca and Bristol Myers Squibb merger talks were reported, then denied.

• Curium and Lantheus agreed an $8bn radiopharma deal.

• Supernus and Indivior agreed a $7.5bn all-stock merger

• Moderna secured approval for the first mRNA flu vaccine.

• Latigo and BlossomHill closed a four-IPO week in the US.

Browse by day:

• Monday: Curium-Lantheus · Supernus-Indivior · KKR-Integer · BioNTech · Gilead

• Tuesday: Pfizer · Novo Nordisk · Merck TL1A · Pathos AI · P&G-Thorne

• Wednesday: Takeda US approval · Eli Lilly · Attovia · PacBio · GRAIL · LifeMine

• Thursday: Replimune · Moderna · Tarsus-Alkeus · Braveheart · Intellia · FDA pilot · AI

• Friday: Latigo · BlossomHill

Story of the week

The FT reported that AstraZeneca and BMS had held talks about a $400bn merger which if true would create the world’s fourth-largest drugmaker by value

The weekend report triggered sharp market reactions on Monday, despite neither company issuing official comment. AZ shares fell as much as 9% in early trading, its worst move since 2020, while BMS gained 6% in US pre-market trading.

In a Reuters article on Wednesday, an insider dismissed the speculation entirely. “There is no deal between AstraZeneca and BMS. There never was a deal to be done, and there are no discussions between the companies,” the source stated.

• AstraZeneca holds talks with BMS over $400bn tie-up · Financial Times

• Exclusive: ‘No discussions’ over AstraZeneca-BMS deal, senior source says · Reuters

Weekend catch-up

Monday 3 August

Curium agreed to buy Lantheus in a deal worth up to $8bn.

Curium, which is PE backed, will pay $102.50/share in cash plus contingent value rights (CVRs) worth up to $12.00/share on commercial milestones to 2030. The CVRs split $6.00 to prostate cancer diagnostics, $3.00 neurology and $1.00 the DEFINITY imaging agent. The combination would put diagnostic and therapeutic radioligand assets under one owner across more than 70 countries. Expects to close H1 2027.

Curium Announces Definitive Agreement to Merge with Lantheus · Curium

Supernus and Indivior agreed an all-stock merger to create a $7.5bn in value and $2.2bn in revenue central nervous system drugmaker.

Each Supernus share converts into 1.54 Indivior shares, leaving Indivior holders with about 56.5% and Supernus holders 43.5%. Indivior shareholders also take a $1bn special cash dividend before closing, funded partly by a $650m Citibank term loan. The combined company keeps the Supernus name and Jack Khattar as CEO. It targets $888m of adjusted EBITDA including $125m cost synergies. Expects to close Q4 2026.

• Supernus and Indivior to Merge · Supernus Pharmaceuticals

KKR is taking US device maker Integer private at an enterprise value of $5.7bn.

Integer shareholders get $127.00/share in cash, 51.8% above the 29 April close, the day before it announced a strategic review, and 28.8% above the 30-day volume-weighted price to 31 July. There is no financing condition. Expects to close end 2026

Integer to Be Acquired by KKR in Transaction Valued at $5.7 Billion · Integer Holdings

BioNTech named Sobi chief executive Guido Oelkers as its next chief executive.

Oelkers takes office by 1 February 2027 and succeeds co-founder Ugur Sahin, who leaves with CMO Ozlem Tureci to run a new next-gen mRNA company. Oelkers more than quadrupled revenue at Sobi over 2017 to 2026. He will prioritise BioNTech’s evolution into a global biopharma firm with multiple approved products by 2030.

• Appointment of Guido Oelkers to Board as Chief Executive Officer · BioNTech

The Supreme Court of California ruled that drugmakers owe no duty to bring a safer alternative to market sooner, ending class action litigation against Gilead.

Justice Groban wrote manufacturers “do not owe a duty of care to users of a nondefective drug when making decisions about whether and when to commercialize an allegedly safer alternative.” The plaintiffs took Viread and argued Gilead had held back Vemlidy to protect revenue. It reverses and earlier Court of Appeal decision.

• Gilead Tenofovir Cases, S283862 · Supreme Court of California

Tuesday 4 August

Pfizer added $2.5bn to its cost-cutting programme.

The new reductions run from 2027 to 2029 and take targeted net savings to $9.7bn through 2029. Q2 revenue was $15.0bn, up 1%, with adjusted earnings of $0.77 a share. Pfizer lifted its 2026 revenue guidance by $500m to $60.5-$62.5bn.

Pfizer Reports Q2 Results And Raises Midpoint of 2026 Revenue Guidance · Pfizer

Novo Nordisk raised its 2026 guidance driven by GLP-1 volumes.

Adjusted sales grew 7% at CER to DKK 78.5bn and adjusted operating profit 11% to DKK 33.4bn. Full-year adjusted sales and operating profit growth is now guided at 0% to -6%, against -4% to -12% before. The Wegovy pill has passed 5m prescriptions since launch and was running above 265,000 a week in mid-July.

• Novo Nordisk reports Q2 2026 and raises full-year outlook · Novo Nordisk

Merck’s anti-TL1A antibody tulisokibart met its endpoints in hidradenitis suppurativa and missed in a rare lung disease.

The systemic sclerosis-associated interstitial lung disease programme is discontinued, and the full hidradenitis results go to a medical meeting. Merck paid $10.8bn for Prometheus Biosciences to acquire the antibody. Q2 sales were $16.6bn, up 5%, with $8.4bn from Keytruda and a $2.31/share charge tied to the Terns Pharma purchase.

Q2 2026 Financial Results; Key Regulatory & Clinical Milestones · Merck & Co

Pathos AI licensed Alphamab Oncology’s TROP2/HER3 bispecific antibody-drug conjugate in a deal worth up to $2.2bn.

Pathos pays $125m upfront for rights outside mainland China, Hong Kong, Macau and Taiwan, with up to $2.09bn in milestones and tiered royalties. JSKN016 is already in phase 3 in triple-negative breast cancer in China. Pathos also named AZD4241, an oestrogen-receptor PROTAC licensed from AstraZeneca, in its pipeline.

• Global Licensing Agreement with Alphamab Oncology on JSKN016 · Pathos AI

P&G agreed to buy US supplements maker Thorne from L Catterton for $3.8bn.

L Catterton bought into the vitamins, minerals and supplements firm in October 2023.

Sale of Thorne to The Procter & Gamble Company for $3.8 Billion · L Catterton

Wednesday 5 August

Takeda won US approval for the first orexin agonist for narcolepsy.

The FDA cleared Orzeyful, an oral orexin receptor 2 agonist, for narcolepsy type 1 in adults. It is the first disease modifying medicine directed at orexin deficiency and improved excessive daytime sleepiness, cataplexy and quality of life in phase 3 trials.

• FDA Approves ORZEYFUL for Adults With Narcolepsy Type 1 · Takeda

Eli Lilly raised guidance as tirzepatide neared two-thirds of revenue.

Q2 revenue was $23.0bn, up 48%: Mounjaro (tirzepatide for type 2 diabetes) took $9.9bn, up 91%, and Zepbound (for weight loss) $4.9bn, up 46%. The newly launched oral GLP-1 Foundayo (orforglipron) took $98m. Lilly raised 2026 revenue guidance +3% to $85-$87bn from $82-$85bn and narrowed EPS guidance to $35.50-$36.50.

• Lilly reports second-quarter 2026 financial results, raises full-year guidance · Eli Lilly

Attovia Therapeutics priced an upsized $289m Nasdaq IPO.

The company sold 17m shares at $17.00 and began trading as ATTO. Proceeds fund three immunology candidates, led by the anti-IL-31 antibody ATTO-1310 in chronic itch, which is positioned against Sanofi and Regeneron’s Dupixent (dupilumab).

• Pricing of Upsized Initial Public Offering · Attovia Therapeutics

PacBio replaced its chief executive, cut its revenue guidance and restructured.

COO Mark Van Oene became president and CEO with immediate effect, succeeding Christian Henry after six years; Henry stays on the board and will advise to end 2026. 2026 revenue guidance fell $10m (6%) to $155-$165m. Q2 revenue was $39.0m vs $39.8m, the net loss widened to $44.7m, and cash fell to $236.9m from $314.7m.

• PacBio Second Quarter 2026 Financial Results · Pacific Biosciences

• PacBio Appoints Mark Van Oene as President and CEO · Pacific Biosciences

GRAIL expects an FDA advisory committee this autumn on Galleri, its multi-cancer early detection (MCED) blood test.

The premarket approval application rests on 25,000 PATHFINDER 2 participants with a year of follow-up and on the first screening round of the 140,000-participant NHS-Galleri trial in England. Q2 revenue rose 26% to $44.7m and Galleri volume 35% to >61,000 tests, against a net loss of $110.2m and cash of $861.6m. GRAIL also closed a $110m equity financing with Samsung to sell Galleri in South Korea.

• GRAIL Reports Second Quarter 2026 Financial Results · GRAIL

LifeMine raised a $188m series E to take transplant drug into later trials.

A $75m series D from Q4 2025 and an oversubscribed $188m series E led by Milky Way Investments, with Bezos Expeditions, Gates Frontier, RA Capital, GV and ARCH Venture Partners, fund LIFE-001. The molecule came out of mining fungal genomes, is given as a long-acting injection and does not enter the brain.

• $263m raise to Accelerate Clinical Development of LIFE-001 · LifeMine Therapeutics

Thursday 6 August

Stanford and Arc Institute scientists designed 16 viable bacteriophages with AI.

Writing in Science, the team used the Evo genome language model to generate whole bacteriophage genomes letter by letter, synthesised hundreds of candidates and found 16 that were viable. All 16 infect E. coli and pose no threat to humans. A cocktail of them overcame bacteria that had resisted a natural phage. The research highlights the need for biosecurity governance guardrails to be properly established.

• Generative design of bacteriophages with genome language models · Science

FDA approved Replimune’s Tudriqev for advanced melanoma at the 3rd attempt.

The accelerated approval covers Tudriqev with Opdivo in unresectable advanced cutaneous melanoma that has progressed on an anti-PD-1 regimen. About a quarter of evaluable patients in the single-arm trial responded, for a median of just over 14 months. Continued approval depends on the confirmatory phase 3 IGNYTE-3 trial.

• FDA Accelerated Approval of TUDRIQEV with Nivolumab · Replimune

Moderna won US approval for the first mRNA flu vaccine.

mFLUSIVA (mRNA-1010) is cleared for adults 50 and over: a standard approval for those 50-64 based on a 40,805-participant phase 3 in 11 countries, and accelerated approval for those 65 and over based on immunogenicity data from 2,992 US adults, with a confirmatory study required. This reverses a refusal to file the agency issued on the same application earlier this year. Supply is expected for the 2026-27 season.

• Moderna Receives U.S. FDA Approval for Influenza Vaccine mFLUSIVA · Moderna

Tarsus Pharmaceuticals agreed to buy Alkeus Pharmaceuticals for $450m up front, taking it into inherited retinal disease.

Tarsus pays $270m in cash and $180m in stock, with up to $350m on regulatory approval and first commercial sale plus tiered royalties. Alkeus’s gildeuretinol is in the 230-patient phase 3 NORTHSTAR trial in Stargardt disease, which affects >36,000 Americans and has no approved treatment; topline data are due in H2 2029.

Tarsus to Acquire Alkeus and Advance Eye Care Leadership · Tarsus Pharmaceuticals

Braveheart Bio priced an upsized $382.5m IPO above its range.

The company sold 21.25m shares at $18.00, against a marketed $15 to $17, and began trading as BRVE. Proceeds fund phase 3 work on a cardiac myosin inhibitor licensed from Jiangsu Hengrui for hypertrophic cardiomyopathy, against BMS’s Camzyos.

• Braveheart Bio Announces Pricing of Upsized Initial Public Offering · Braveheart Bio

Intellia linked the liver enzyme spikes in its CRISPR trials to a single HLA allele.

A genomic analysis of >600 patient samples across the nex-z programme found the highest transaminase elevations in carriers of one HLA allele. Intellia is discussing the finding with regulators and will screen patients entering its phase 3 trials.

• Intellia Announces Q2 2026 Results and Business Updates · Intellia Therapeutics

The FDA set out how its proposed expedited investigational new drug (IND) pilot would work.

Qualified research institutions, CROs and other third parties would help sponsors prepare and pre-review IND material on a rolling basis, shortening the time from picking a molecule to a first-in-human trial. The FDA would keep full authority over regulatory decisions, including the power to place a trial on clinical hold.

• Expedited IND Pilot Program Educational Webinar for Stakeholders · FDA

• Expedited IND Pilot Program; Request for Information · Federal Register

Friday 7 August

Latigo and BlossomHill priced IPOs to close four biotech IPOs this week.

Latigo Biotherapeutics sold 19.2m shares at $18.00 for $345.6m, funding LTG-001, an oral NaV1.8 painkiller positioned against Vertex Pharmaceuticals’ Journavx.

BlossomHill Therapeutics sold 9.375m shares at $16.00 for $150m, funding: BH-30643 in EGFR-mutant lung cancer, a would-be rival to AstraZeneca’s Tagrisso; and BH-30236 in acute myeloid leukaemia and higher-risk myelodysplastic syndrome.

Both begin trading today, taking the week’s US biotech IPOs to four and $1.2bn raised.

• Latigo Announces Pricing of Upsized $345.6m IPO · Latigo Biotherapeutics

• BlossomHill Announces Pricing of Upsized $150m IPO · BlossomHill Therapeutics

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines

• Biopharma dealmaking hit a record $170bn in H1 2026.

• GSK chose Cambridge for its new R&D HQ.

• Novo Nordisk‘s ziltivekimab failed its heart trial.

• Johnson & Johnson offered $5.5bn to settle talc claims.

• argenx agreed to buy Forte Biosciences for $2.2bn.

In this edition

• Monday: argenx-Forte deal; AstraZeneca Q2; Otsuka ADHD approval; Freenome-Abbott test; J&J talc settlement

• Tuesday: GSK move to Cambridge; US Medicare Part D

• Wednesday: UK social care; FTC v Hims; J&J-Sail CAR-T; Biogen Leqembi; Capricor DMD vote; Latigo pain; record biopharma M&A

• Thursday: Sanofi cleanup; Replimune adcomm; Alnylam cut; US pharma tariffs; decongestant suit; Illumina guidance; biotech IPOs

• Friday: Novo ZEUS failure; Datroway EU approval

Monday 27 July

argenx agreed to buy Forte Biosciences for $2.2bn, gaining a first-in-class antibody in vitiligo and coeliac disease.

The Dutch-Belgian immunology group is paying $77 a share in cash, an 86% premium to Forte’s volume-weighted price since it reported positive phase 1b vitiligo data on 9 July. It gains FB102, an anti-CD122 antibody with early proof-of-concept in vitiligo and coeliac disease, with phase 2 readouts due in H2 2026. The deal carries argenx beyond the neuromuscular and autoantibody diseases it built Vyvgart (efgartigimod) around, into two indications with no established drug.

• argenx to Acquire Forte Biosciences, Adding First-in-Class anti-CD122 FB102

AstraZeneca posted an overall-survival win for its Claudin 18.2 drug in gastric cancer and a rare-disease miss for Ultomiris, alongside Q2 results.

AZ said sonesitatug vedotin met the overall-survival primary endpoint in later-line CLDN18.2-positive advanced gastric cancer and a key secondary endpoint in the broader population, although PFS missed significance. Ultomiris failed on event-free survival in transplant-associated thrombotic microangiopathy. Management retained its $80bn 2030 revenue target.

• Half-year Financial Report - AstraZeneca (RNS)

• Update on Phase III trial of Ultomiris in adults and adolescents with thrombotic microangiopathy after haematopoietic stem cell transplant - AstraZeneca

The FDA approved Otsuka’s Simtriyo (centanafadine), the first triple reuptake inhibitor for ADHD.

Otsuka announced the approval on 27 July for adults and children aged six and over, three days after the agency cleared it. Simtriyo blocks reuptake of norepinephrine, dopamine and serotonin, a mechanism no approved ADHD drug uses, and must clear controlled-substance scheduling before it can launch.

• Otsuka Receives FDA Approval for First-in-Class SIMTRIYO for Treatment of ADHD

The FDA approved Freenome’s SimpleScreen blood test for colorectal cancer, with Abbott to sell it in the US.

The approval turns SimpleScreen from a lab service into a product Freenome can sell. Under a 2025 agreement, Abbott’s diagnostics operation will commercialise SimpleScreen from this autumn, triggering a $100m milestone for Freenome. In the >48,000-patient PREEMPT CRC study, the test detected colorectal cancer with 81% sensitivity and showed 90% specificity for advanced neoplasia. The test offers a blood-draw alternative to colonoscopy and stool testing, which many patients avoid.

FDA Approves Freenome’s SimpleScreen CRC Blood-Based Screening Test; Abbott to Commercialize in the U.S.

Johnson & Johnson offered $5.5bn to settle ovarian-cancer talc litigation.

The proposal covers roughly 76,000 remaining ovarian-talc claims and requires participation by firms representing at least 95% of them. J&J would commit $5.5bn, with a first payment of no more than $3bn in 2027 and nothing further before 2028. It lands below the $8.9bn J&J proposed in 2023 and follows two failed attempts to route the claims through bankruptcy. A parallel case runs in the UK.

• Johnson & Johnson Announces a Proposed Resolution of Ovarian Talc Litigation

Tuesday 28 July

GSK will move its R&D HQ to Cambridge and cut £1.9bn annual cost by 2029.

The Cambridge Biomedical Campus centre replace GSK’s Stevenage site and house >1,000 scientists across oncology, respiratory, hepatology, vaccines and HIV. GSK targets £1.9bn in annual savings for £2.4bn of costs over three years, mainly through procurement, simpler processes and shifting resources from mature products. It now plans 20+ phase 3 starts in 2026, double its previous target, and booked a £1.3bn impairment, mostly on chronic-cough drug camlipixant. Q2 turnover was £8.4bn.

• GSK delivers strong Q2 core results performance and continued momentum

CMS will end subsidy holding down standalone Medicare drug-plan premiums.

Centers for Medicare & Medicaid Services (CMS) said it will close the Part D Premium Stabilization Demonstration at the end of 2026, having judged that from 2027 bids that insurers can now price plans without the support. The programme has subsidised standalone Part D plans across 2025 and 2026 to blunt premium rises, so its removal exposes enrollees to sharper increases next year.

• July 28, 2026 Medicare Parts C & D Announcement - CMS

Wednesday 29 July

Prime Minister Andy Burnham asked the Casey commission to set out how to deliver a National Care Service for England.

In a speech, Burnham announced Baroness Louise Casey’s Independent Commission on Adult Social Care will deliver final recommendations by summer 2027 for a service built around the person and integrated with the NHS. He said care workers should be “the best paid people in society, not the worst“. Casey has argued for a mixed market while criticising some private providers for profiteering. It is the new government’s attempt to reopen a reform successive administrations have left unresolved.

• Prime Minister’s speech on social care: 29 July 2026 - Prime Minister’s Office

The FTC sued Hims & Hers, alleging it shared users’ health data with advertisers after promising to protect it.

The complaint, filed with the State of Utah and Los Angeles County in California federal court, says the telehealth company passed lists of customers identified by condition or treatment to Meta, Snap and others through tracking pixels, and locked subscribers into plans while making cancellation hard. It invokes the FTC Act and the Restore Online Shoppers’ Confidence Act. Hims & Hers, a major telehealth supplier of weight-loss drugs, called the claims baseless.

• FTC & States Act Against Hims & Hers for Deceptive and Unlawful Privacy Practices

J&J took a $2.6bn option to buy in vivo CAR-T company Sail Biomedicines.

J&J will pay $785m up front, including $465m in equity, plus up to $140m in milestones, and $2.58bn more if it exercises the option. Sail, built by Flagship Pioneering, develops RNA medicines that engineer a patient’s T cells inside the body rather than in a manufacturing suite, which is designed to dramatically simplify cell therapy manufacture. Buying an option rather than the company lets J&J hold the position while the data matures.

• J&J Collaboration with Sail Biomedicines to Advance in vivo CAR-T Programs

Biogen raised its 2026 guidance as Leqembi recorded its best quarter.

Global Leqembi (lecanemab) sales, shared with Eisai, rose 15% to $184m, a third straight quarter of growth for an Alzheimer’s antibody held back since approval by its infusion schedule and brain imaging. Q2 revenue of $2.74bn beat consensus, and Biogen improved its full-year revenue outlook from a mid-single-digit decline to an increase. Newer drugs, including the Apellis-acquired Syfovre and Empaveli, now outweigh the declining multiple sclerosis business.

• Biogen Reports Second Quarter 2026 Results

An FDA advisory committee voted against Capricor’s Duchenne cell therapy.

The panel voted 9 to 3 that the efficacy data did not support deramiocel for the heart-muscle damage of DMD. The vote does not bind FDA, which has a 22 August decision date, but a negative vote leaves little room to approve without more evidence.

• Capricor Provides Update on FDA Advisory Committee Meeting for Deramiocel

Latigo’s non-opioid painkiller succeeded in a mid-stage trial, putting a second drug into a field Vertex has had to itself.

Published in the New England Journal of Medicine (NEJM), a phase 2b trial in 343 abdominoplasty patients found LTG-001 reduced 48-hour pain versus placebo. Latigo said the high dose delivered about 50% greater SPID48 than Vicodin, with 52% of patients opioid-free versus 22% on placebo. Vertex secured the class’s first approval with Journavx; a credible rival now complicates the market for payers and partners. Latigo, which is pursuing an IPO, plans phase 3 trials in H2 2026.

• Latigo: NEJM Publication of Positive LTG-001 Abdominoplasty Clinical Trial Results

Biopharma dealmaking hit a record $170bn in the first half of 2026, on fewer but larger transactions.

BioWorld put H1 deal value at $170.1bn, 22% above the $139.4bn of H1 2025 and the highest first half in its records, with the second quarter alone a record $90.4bn. The deal count fell to 579 from 636, so the increase was driven by bigger deals. The patent cliff is a key driver, to shore up big pharma’s next decade of revenue.

• Biopharma shatters quarterly deal record with $90.4B Q2 haul - BioWorld

Thursday 30 July

Sanofi upgraded its 2026 guidance but cleared out late-stage immunology programmes under new chief executive Belen Garijo.

Q2 sales rose 17.8% to €11.6bn, led by Dupixent up 37.6%, and Sanofi lifted full-year guidance to around 10% sales growth. Against that, Garijo discontinued the Regeneron-partnered IL-33 antibody itepekimab in COPD and chronic rhinosinusitis and the balinatunfib programmes, and impaired the Kymab-derived amlitelimab by €952m, part of write-offs of over €1bn. It was her first account of how she means to reverse a run of clinical setbacks: an R&D cleanup, more M&A and a push into China.

• Q2 26: double-digit sales and strong EPS growth; 2026 guidance upgraded - Sanofi

An FDA adcom backed Replimune’s twice-rejected melanoma drug, 10 to 3.

The panel found the efficacy of RP1 (vusolimogene oderparepvec) with Opdivo clinically meaningful in melanoma that has progressed on anti-PD-1 therapy, days after FDA reviewers said in briefing documents that the registrational IGNYTE trial did not prove RP1 works. The vote does not bind FDA, which has a 2 August decision date.

• Replimune Announces Favorable Outcome of FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee Meeting for RP1 in Advanced Melanoma

Alnylam cut its TTR sales outlook as Amvuttra hit a $1bn+ quarter for first time.

TTR revenue reached $1.03bn, up 89%, taking total product sales to $1.17bn, but Alnylam trimmed full-year TTR guidance by $200m to $4.2 to $4.5bn, saying early second-line demand had been inflated by pent-up switching that has now normalised. Amvuttra (vutrisiran) makes up most of Alnylam’s commercial weight.

• Alnylam Pharmaceuticals Reports Second Quarter 2026 Financial Results

Endpoints reported that the first round of Trump’s 100% pharmaceutical tariffs will not take effect as planned, every targeted company having dealt itself out.

The first Section 232 tariffs were due to hit an initial group of drugmakers, but all had secured Most Favoured Nation pricing and US onshoring deals setting their rate at 0%, leaving none to charge. The 100% tariff remains scheduled for the rest of the industry from 29 September, as the White House presses more firms into agreements.

• 1st round of Trump’s 100% pharma tariffs won’t start Friday as planned - Endpoints

A US appeals court revived claims that decongestant makers misled buyers with “maximum strength” labelling.

The Second Circuit ruled that “maximum strength“ claims over phenylephrine products can proceed. The FDA has concluded that oral phenylephrine works no better than placebo. The consolidated litigation covers roughly 100 class actions against makers and retailers including Procter & Gamble and CVS.

• In re Oral Phenylephrine Marketing and Sales Practices Litigation, No. 24-3296 (2d Cir.) - US Court of Appeals for the Second Circuit

Illumina raised its 2026 guidance after a Q2 beat.

The sequencing leader reported Q2 revenue of $1.16bn, up 9.5%, and lifted full-year guidance to $4.60 to $4.64bn and non-GAAP EPS to $5.30 to $5.40, citing NovaSeq X placements and clinical demand, a read across the entire sequencing market.

• Illumina Reports Financial Results for Second Quarter of Fiscal Year 2026

Two biotechs priced or set terms for IPOs

Apnimed priced an upsized offering of 12m shares at $16 to raise $192m, funding the launch of its sleep-apnoea pill AD109, which awaits an FDA decision, and its shares jumped on their Nasdaq debut. Braveheart Bio set terms to raise $318.8m at a valuation of up to $1.2bn, to fund phase 3 work on BHB-1893, a cardiac myosin inhibitor licensed from Jiangsu Hengrui for hypertrophic cardiomyopathy. Analysts attribute the revival to rotation from richly valued tech and AI stocks into healthcare.

• Apnimed Announces Pricing of Upsized Initial Public Offering

• Braveheart Bio, Inc. Form S-1/A - SEC EDGAR

Friday 31 July

Novo’s ziltivekimab failed in its big cardiovascular outcomes trial, shutting its expansion from obesity and diabetes.

In phase 3 ZEUS, the anti-IL-6 antibody engaged its target but failed to reduce major cardiovascular events versus placebo in >6,300 people with atherosclerotic disease, chronic kidney disease and inflammation (hazard ratio 0.99). Cardiovascular outcome trials are among the costliest and determine reimbursement breadth, so failure leaves little room for recovery. Novo’s remaining hopes rest on two trials: HERMES in heart failure and ARTEMIS after heart attack, both of which report in H1 2027.

• Novo update on ZEUS phase 3 trial in people with ASCVD, CKD and inflammation

AstraZeneca and Daiichi Sankyo won EU approval for Datroway in first-line triple-negative breast cancer.

The European Commission cleared Datroway, a TROP2 ADC, as first-line monotherapy for metastatic triple-negative breast cancer in patients ineligible for immunotherapy, moving it ahead of chemotherapy. In phase 3 TROPION-Breast02, overall survival rose to 23.7 months from 18.7 (median) and the risk of progression or death fell 43%. AZ said it is the only EU-approved TROP2 with an overall-survival benefit in this setting.

• Datroway approved in the EU as only TROP2-directed medicine with overall survival benefit for 1st-line metastatic TNBC - AstraZeneca

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines

• Burnham abolished the UK science department.

• Biotech M&A hit a new record.

• Trump threatened 200% tariffs on imported generics.

• Tempus AI agreed to acquire Personalis for $1.5bn.

• J&J gained FDA approval for its first robotic surgery system.

In this edition

• Monday: Samsung buys PolyPeptide; GSK drops camlipixant; Kolon phase 3 fail; Tempus buys Personalis

• Tuesday: UK abolishes DSIT; record biotech M&A; Novartis-Myricx ADC; Freenome SPAC

• Wednesday: Trump generic tariffs; Novo sues Lilly; GSK Jideytro approval; Crystalys gout raise; NIH Bio Genesis

• Thursday: Repligen buys BioLife; AstraZeneca EU breast cancer; J&J robotic surgery; Lilly retatrutide

• Friday: FDA backs BPC-157; J&J myeloma combo; GSK strategy update

Monday 20 July

Samsung Biologics agreed to buy Swiss CDMO PolyPeptide Group for $1.8bn.

Samsung is offering CHF 44.31 a share in cash, valuing PolyPeptide’s equity near CHF 1.46bn ($1.8bn), a 40% premium to the undisturbed price on 10 April. PolyPeptide’s board backs the offer and its largest holder, with 55.7% of the shares, has committed to tender. Peptide contract manufacturers have become a choke point in weight-loss drugs. Samsung, until now a monoclonal-antibody house, is buying its way into that. Completion is expected by the end of 2026.

• Samsung Biologics announces tender offer to acquire PolyPeptide Group AG for CHF 44.31 per share - PolyPeptide Group / Samsung Biologics

GSK is stopping development of chronic-cough drug it paid $2bn for.

In the phase 3 CALM programme, CALM-1 hit its primary endpoint, a significant cut in 24-hour cough frequency at week 12 on the 50mg twice-daily dose, but CALM-2 missed the same endpoint at week 24 and the lower 25mg dose failed in both trials. GSK judged the benefit “unlikely to transform patient care” and stopped development in refractory chronic cough. The asset, a P2X3 antagonist it acquired in its 2023 Bellus Health buyout, continues in a phase 2b trial in IBS.

• GSK provides an update on CALM-1 and CALM-2 phase III trials - GSK

Kolon’s gene therapy for knee osteoarthritis failed its first late-stage US trial.

TissueGene-C (TG-C) missed both co-primary endpoints, pain and function, at month 12 in the phase 3 ACTiVION-II study, which the company put down to an unusually strong placebo response. A second late-stage trial, ACTiVION-I, reports in October.

• Kolon TissueGene reports topline ACTiVION-II phase 3 results for TissueGene-C (TG-C) - Kolon TissueGene

Tempus will buy Personalis for $1.5bn adding cancer genomics to its AI platform.

The all-stock deal values Personalis at $16.25 a share, an enterprise value near $1.5bn net of Tempus’s existing stake, with Tempus able to pay up to half in cash. It folds Personalis’s tumour-profiling and molecular residual disease (MRD) testing, including its NeXT Personal assay, into Tempus’s precision-oncology business. Tempus expects to close late in 2026 or early in 2027.

• Tempus to acquire Personalis, integrating MRD - Tempus AI

Tuesday 21 July

New PM Burnham cut the science department and established a Minister for AI.

The Department for Science, Innovation and Technology (DSIT) is being folded into an enlarged Department for Business, Innovation, Science and Trade under Jonathan Reynolds. Patrick Vallance, the former government chief scientific adviser, resigned as science minister. Life sciences leaders welcomed Reynolds’s return but voiced concern at losing both a standalone science department and Vallance. A new Minister for AI, Kanishka Narayan, gets a cabinet seat, with AI strategy moving into the Cabinet Office.

• Ministerial appointments: July 2026 - Prime Minister’s Office (GOV.UK)

• Departure of Lord Vallance as science minister causes concern - The Times

Billion-dollar biotech takeovers in 2026 have already passed the whole of 2025.

The Financial Times counted 37 acquisitions of $1bn or more so far in 2026, topping last year’s full-year record of 35 with five months still to run. The engine is the patent cliff: big pharma is sitting on cash from today’s blockbusters and buying its next decade of revenue. The 2026 trend is broad, with every major and a widening group of mid-sized and specialist buyers in the market.

• Fervour over top-selling drugs fuels record biotech dealmaking - Financial Times

Novartis provided further colour on its $1.5bn Myricx Bio acquisition.

Chief executive Vas Narasimhan defended the deal on this week’s Q2 call. Struck earlier in July, it brings Novartis Myricx’s N-myristoyltransferase inhibitor payload, a different way of killing tumour cells from the ADCs already crowding oncology. Narasimhan, who had sat out the ADC land grab, said he moved because the chemistry was novel: “we would certainly look to expand that NMTi payload across more delivery vehicles and more drug targets.”

• Novartis agrees to acquire Myricx Bio, advancing next-generation ADC innovation with a novel NMTi payload - Novartis

• “We’ve been looking for a novel approach”: Why Novartis CEO ended his ADC holdout for the $1.5B Myricx deal - Fierce Biotech

Freenome went public through a $310m SPAC.

The multi-cancer early detection (MCED) company completed its combination with Perceptive Capital Solutions Corp and began trading on Nasdaq as FRNM, raising $310m gross. The listing, agreed with the Perceptive Advisors vehicle in December, hands one of the larger private names in liquid-biopsy screening public-market capital to push its colorectal and multi-cancer tests toward commercialisation.

• Freenome debuts as a publicly traded company focused on blood-based early cancer detection - Freenome

Wednesday 22 July

Trump set a countdown to a 200% tariff on imported generic drugs

Generics escape duty for two years, then face a 100% tariff from August 2028 and 200% a year after that, unless their makers build in the US. The president cast it as reshoring “with a penalty to those Companies that decide not to build.” The exposure is real: generics are about 90% of US scripts and most are imported, >50% from India alone. Sandoz, among the largest suppliers, said it shares the affordability goal and would keep talking to Washington but that it was “too early to assess” the impact.

• Truth Social post on a 200% tariff on imported generic drugs - Donald J. Trump, Truth Social

• Sandoz to engage with US policymakers after Trump’s tariff plan - The Wall Street Journal

Novo Nordisk took Eli Lilly to court over false advertising of weight-loss drugs.

In a complaint filed in New Jersey federal court, Novo alleges Lilly ran “false and materially misleading“ ads for Zepbound and Mounjaro, “intentionally selected outdated studies,” and set its top doses against lower doses of Wegovy and Ozempic while leaving out newer high-dose semaglutide data. It wants an injunction and corrective advertising. Lilly’s campaign leans on the SURMOUNT-5 head-to-head trial where tirzepatide beat semaglutide on weight loss: “the only head-to-head, randomized clinical trial“.

• Novo Nordisk v. Eli Lilly, complaint, D.N.J. (3:26-cv-09027) - Novo Nordisk / STAT News

The FDA approved GSK’s cancer drug Jideytro, a week after its $10.6bn buyout.

Jideytro (zidesamtinib), a brain-penetrant ROS1 inhibitor, cleared for ROS1-positive non-small cell lung cancer in patients already treated with a ROS1 drug, nearly two months ahead of its September action date. It is GSK’s first approved lung-cancer medicine, and it arrived through Nuvalent, whose ~$10.6bn takeover GSK completed on 15 July. Approval rested on the phase 1/2 ARROS-1 trial, in which 44% of patients responded.

• Jideytro (zidesamtinib) approved in the US for previously treated ROS1-positive NSCLC - GSK

• GSK completes acquisition of Nuvalent, Inc. - GSK

Crystalys raised $130m to push its once-daily gout pill into phase 3.

The Series B, led by Frazier Life Sciences alongside existing backers Novo Holdings and SR One, funds registration trials for dotinurad, an oral URAT1 inhibitor that lowers uric acid and is already approved across much of Asia. It follows a $205m Series A only ten months earlier, in September 2025.

• Crystalys Therapeutics announces $130 million Series B financing to advance global phase 3 development of dotinurad for gout - Crystalys Therapeutics

The NIH put its weight behind AI, launching a $1.2bn Bio Genesis Mission.

The programme is the health agency’s slice of a wider federal Genesis Mission, set up by a November 2025 executive order to point AI at scientific problems across government. NIH names six targets, from forecasting biological systems to speeding drug development, and its director, Jay Bhattacharya, set a goal of halving the time from discovery to patient over ten years.

• Genesis Mission: harnessing AI to transform American science - The White House

Thursday 23 July

Repligen agreed to buy BioLife Solutions for about $1.5bn

BioLife makes the biopreservation media and freezing products that keep cell and gene therapies alive from manufacturing to patient, so the deal moves Repligen further into that supply chain rather than into a drug. Terms are $31.00 a share, split $11.25 in cash and 0.1442 of a Repligen share, a 24% premium to the 90-day average, with close expected in Q4.

• Repligen to acquire BioLife Solutions, expanding its cell-therapy capabilities - Repligen / BioLife Solutions

AstraZeneca won EU approval for its oral breast-cancer pill Etcamah

Etcamah (camizestrant), a next-generation oral selective estrogen receptor degrader (SERD), is cleared with a CDK4/6 inhibitor for ER-positive, HER2-negative advanced breast cancer once a tumour picks up an ESR1 mutation during first-line treatment. In the phase 3 SERENA-6 trial the combination cut the risk of progression or death by 56% (hazard ratio 0.44), lifting median progression-free survival to 16.0 months from 9.2. The FDA has extended its review in the US approval process.

• Etcamah (camizestrant) approved in the EU for ESR1-mutant advanced breast cancer - AstraZeneca

J&J was cleared by the FDA to enter the surgical robotics market.

The agency granted De Novo authorisation to J&J’s Ottava system, a soft-tissue surgical robot with its arms built into the operating table rather than mounted on carts, for a broad set of general-surgery procedures. It sets up a three-way US contest with Intuitive Surgical‘s da Vinci, which holds about 80% of the market, and Medtronic‘s Hugo. J&J will start with selected US hospitals while it seeks more indications.

• Johnson & Johnson receives FDA market authorization in the US for its OTTAVA Robotic Surgical System - Johnson & Johnson

Lilly’s retatrutide posted the largest phase 3 weight loss yet reported for an obesity drug, but did not significantly reduce cardiac events.

In two phase 3 trials, the triple hormone agonist (”triple-G”) took up to 22.6% off patients’ weight at 80 weeks, ahead of the GLP-1 drugs already on sale. But in TRIUMPH-3, run in people with established cardiovascular disease, it did not significantly cut major cardiac events, the hazard ratio of 0.82 too wide to tell apart from chance. The peak figure also came in below the roughly 24% seen in phase 2. Lilly plans to file for approval in Q1 2027.

• Lilly’s triple-agonist retatrutide successful in two additional phase 3 trials - Eli Lilly

Friday 24 July

An FDA advisory panel voted to loosen the rules on a barely characterised peptide, over its own scientists’ objections.

By 8 votes to 6, with one abstention, the Pharmacy Compounding Advisory Committee recommended adding BPC-157, alongside a second peptide, KPV, to the substances pharmacies may compound. FDA staff had advised against, citing a lack of evidence that BPC-157 works and a warning of low confidence in what the substance even is. Most of the members who voted yes have ties to the peptide industry, on a panel remade under Health Secretary Robert F. Kennedy Jr. The recommendation is non-binding.

• Certain bulk drug substances for use in compounding that may present significant safety risks - FDA

J&J reported that pairing two of its bispecific antibodies cut the risk of myeloma getting worse by 89%.

In the phase 3 MonumenTAL-6 trial, the combination of Tecvayli (teclistamab) and Talvey (talquetamab), which hit two different targets, BCMA and GPRC5D, reduced the risk of disease progression or death by 89% (hazard ratio 0.11) and the risk of death by 62% against standard care, in patients with relapsed or refractory disease. J&J said the hazard ratio was the lowest yet reported for a bispecific combination in a phase 3 myeloma trial. The result strengthens J&J’s already leading myeloma franchise.

• Tecvayli and Talvey reduced the risk of disease progression or death by 89% in earlier-line relapsed/refractory multiple myeloma - J&J

GSK’s new CEO, Luke Miels, will set out his strategy next week.

The company has scheduled its Q2 results and a “portfolio growth opportunities” update in London for 28 July, and insiders expect the depth of the R&D pipeline, and an emerging oncology portfolio, to be the centrepiece. Miels, previously chief commercial officer, has already spent to reshape that pipeline, through the acquisitions of RAPT and Nuvalent.

• GSK chief Luke Miels to host first major strategy update - The Times

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• CagriSema lost to Zepbound head-to-head.

• Gilead agreed to buy Arcellx for $7.8bn.

• The FDA proposed a pathway for bespoke genetic medicines.

• Roche unveiled its $150 human genome Axelios instrument platform at AGBT.

• Novo Nordisk will cut US semaglutide list prices to $675.

Browse by day:

• Weekend catch-up: US tariffs

• Monday: CagriSema · Arcellx · FDA pathway · Gossamer · Merck · BioMarin · Ultima

• Tuesday: Novo prices · Braftovi · US vaccine suit

• Wednesday: GSK-35Pharma · HIV pills

• Thursday: Lilly pill · argenx · BMS ADC · Aicuris · Hernexeos · Generate IPO

• Friday: Illumina · Oxford Nanopore

Weekend catch-up

The US Supreme Court struck down Trump’s global tariffs, while pharma escaped the replacement.

The court ruled 6-3 that the International Emergency Economic Powers Act does not give the president authority to impose tariffs. The White House responded the same day with a temporary 10% import surcharge under section 122 of the Trade Act, but pharmaceuticals and APIs were exempted. Drugmakers had spent the prior year restructuring supply chains around the now-invalidated regime. The immediate threat has receded, but the authority to target pharma through other statutes survives.

• Learning Resources v Trump, slip opinion of 20 February · Supreme Court

• Imposing temporary import surcharge, presidential proclamation · The White House

Monday 23 February

Novo Nordisk’s CagriSema failed its head-to-head with Zepbound.

In the open-label REDEFINE 4 trial Novo’s CagriSema - a combination of cagrilintide, an amylin analogue, and semaglutide - failed to show non-inferiority on weight loss versus Lilly’s single-molecule dual GIP/GLP-1 agonist Zepbound (tirzepatide) 15 mg at 84 weeks. CagriSema delivered 20.2% weight loss against 23.6% for tirzepatide.

CagriSema was Novo’s best shot at closing the efficacy gap with Zepbound, and the head-to-head result leaves Lilly clearly ahead for now. A higher-dose CagriSema study starts in H2 2026, with REDEFINE 11 reporting H1 2027. An FDA decision on the filed dose is expected by late 2026. Novo shares fell >12% in early Copenhagen trading.

• REDEFINE 4 head-to-head trial, primary endpoint not achieved · Novo Nordisk

Gilead agreed to buy Arcellx for $7.8bn.

Gilead will pay $115/share in cash plus a $5 contingent value right, converting its 2022 anito-cel collaboration into full ownership and eliminating its existing profit share, milestones and royalties. Arcellx’s BCMA CAR-T is already under FDA review in fourth-line multiple myeloma, with a decision due by 23 December 2026. The deal gives Gilead full control of one of the leading challengers to J&J’s Carvykti and adds another major asset to a cell therapy franchise led by Yescarta and Tecartus.

• Gilead to acquire Arcellx to maximize long-term potential of anito-cel · Gilead

The FDA proposed a pathway for bespoke genetic medicines.

The Plausible Mechanism Framework for individualised therapies targets genetic diseases with a known biological cause. Rather than follow conventional development, the FDA would allow evidence on bespoke treatments from related products, mechanistic understanding and prior manufacturing experience to support approval.

The framework formalises the kind of case-by-case approach used for therapies such as the custom CRISPR treatment given to baby KJ. If it works as intended, it could materially shorten development timelines for ultra-rare genetic diseases.

• Plausible Mechanism Framework to develop individualized therapies · FDA

Gossamer Bio’s seralutinib failed its phase 3 in pulmonary arterial hypertension.

The inhaled PDGFR inhibitor improved six-minute walk distance by a placebo-adjusted 13.3 metres at week 24, but missed the trial’s prespecified statistical threshold despite a nominal p=0.0320. Seralutinib had been positioned as the nearest challenger to Merck & Co’s Winrevair. Gossamer shares fell 77%. Enrolment in the follow-on SERANATA study was paused, and the company plans to discuss a path forward with the FDA. Chiesi co-develops the drug.

• Gossamer Bio announces topline results from the phase 3 PROSERA study · Gossamer Bio

Merck is splitting its human health business ahead of the Keytruda patent cliff.

Oncology will become a standalone division under Jannie Oosthuizen, while a separate specialty, pharma and infectious diseases unit will be led by Brian Foard, joining from Sanofi. Keytruda generated $31.7bn of Merck’s $58.1bn pharma sales in 2025 and loses US exclusivity in 2028. The new structure gives oncology its own leadership while focusing on the portfolio that must replace Keytruda’s earnings.

• Merck evolves operating structure to support portfolio execution · Merck & Co

Astellas took co-rights to Vir’s masked T-cell engager for prostate cancer in a deal worth up to $1.7bn.

Vir gets $335m upfront and near-term milestones for VIR-5500, a dual-masked PSMA T-cell engager in phase 1 prostate cancer. The companies split US profit and loss equally, with Vir eligible for another $1.37bn plus ex-US royalties. The deal funds Vir’s oncology push and gives Astellas another prostate cancer asset alongside Xtandi.

• Astellas & Vir announce global strategic collaboration VIR-5500 · Vir Biotechnology

BioMarin is pulling its haemophilia A gene therapy Roctavian from the market.

The withdrawal follows October’s decision to seek a buyer. None emerged, and BioMarin says the move is unrelated to efficacy or safety. Approved in Europe in 2022 and the US in 2023 at a $2.9m list price, Roctavian never achieved expected uptake, highlighting that one-time gene therapies with imperfect durability are commercially difficult at multi-million-dollar prices, particularly where existing treatment is strong.

• BioMarin voluntarily withdraws Roctavian from the market · BioMarin

Ultima Genomics launched its UG200 sequencing series at AGBT.

The UG200 and UG200 Ultra run the company’s Solaris 2.0 workflows, with the Ultra targeting more than 60,000 30x genomes a year. US pricing starts at $850,000, with shipments due from Q2. Ultima is pushing further into the high-throughput sequencing market where Illumina has historically been strongest.

• Next generation sequencing at scale with UG200 Series launch · Ultima Genomics

Tuesday 24 February

Novo Nordisk will reset US semaglutide list prices to $675.

From January 2027, Wegovy, Ozempic and Rybelsus will all list at $675 a month, cutting Wegovy’s sticker price by half. Net prices are already substantially lower after rebates, so the immediate revenue effect may be limited, but the move further dismantles the old $1,000-plus per month US GLP-1 pricing model.

• Significant reduction US list price for Wegovy, Ozempic and Rybelsus · Novo Nordisk

Pfizer’s Braftovi combination won full approval in first-line colorectal cancer.

The FDA converted Braftovi (encorafenib) with Erbitux (cetuximab) and chemotherapy to full approval in BRAF V600E-mutant metastatic colorectal cancer. BREAKWATER showed a 51% reduction in the risk of death, removing the confirmatory-data overhang for the only targeted first-line regimen in this population.

• US FDA grants full approval to Pfizer’s Braftovi combination regimen · Pfizer

15 US states sued the federal government over the childhood vaccines schedule.

The lawsuit challenges the January decision that removed seven vaccines from universal recommendation, including hepatitis A and B, influenza, Covid-19 and RSV, as well as the replacement of ACIP’s membership. The states are asking the court to overturn both, moving the dispute over US vaccine policy into the courts.

• Multistate lawsuit challenging Kennedy vaccine schedule · Arizona Attorney General

Wednesday 25 February

GSK agreed to buy 35Pharma for $950m in cash.

The Canadian biotech’s lead asset HS235 is a clinical-stage activin signalling inhibitor for pulmonary arterial hypertension (PAH) and PH with preserved ejection fraction heart failure. The pathway is validated by Merck’s Winrevair, which sold $1.4bn last year. HS235 data suggest fat-selective weight loss and improved insulin sensitivity. GSK’s move comes two days after Gossamer’s failure thinned the PAH pipeline.

• GSK enters agreement to acquire 35Pharma Inc · GSK

Gilead and Merck both reported late-stage data for new daily HIV pills.

Gilead’s bictegravir/lenacapavir single tablet maintained virological suppression in switch patients in ARTISTRY-1, while Merck reported phase 3 data for its two-drug doravirine/islatravir regimen. Both companies plan regulatory filings, as they try to simplify daily oral therapy even as longer-acting regimens move toward weekly dosing.

• Single tablet bictegravir & lenacapavir maintained virological suppression · Gilead

• Late-breaking data three phase 3 trials evaluating doravirine/islatravir · Merck & Co

Thursday 26 February

Lilly’s orforglipron beat Rybelsus in a head-to-head diabetes trial.

In ACHIEVE-3, orforglipron 36 mg reduced A1C by 2.2% versus 1.4% for Rybelsus 14 mg on the efficacy estimand, with 9.2% weight loss versus 5.3%. On the treatment-regimen estimand, the gap narrowed to 1.9% versus 1.5%. Adverse-event discontinuations were roughly twice as high with orforglipron.

Three days after Zepbound beat CagriSema head-to-head, this compounds a difficult week of direct comparisons for Novo. Orforglipron is filed in >40 countries. As a small molecule it offers Lilly easier manufacturing and supply than peptide GLP-1s.

• Lilly’s oral GLP-1, orforglipron, delivered superior blood sugar control and weight loss compared to oral semaglutide · Eli Lilly

argenx’s Vyvgart succeeded in ocular myasthenia gravis.

The phase 3 ADAPT OCULUS trial met its primary endpoint, with no new safety signals, supporting a US filing to expand Vyvgart into the ocular form of the disease. It is the first registrational study of a targeted treatment in ocular myasthenia gravis.

• Vyvgart phase 3 ADAPT OCULUS data in ocular myasthenia gravis · argenx

BMS and SystImmune’s bispecific ADC hit survival endpoints in breast cancer.

Izalontamab brengitecan, the EGFRxHER3 ADC BMS licensed for $800m upfront in 2023, improved both progression-free and overall survival at an interim analysis of a Chinese phase 3 trial in triple-negative breast cancer. It is the drug’s third positive phase 3 result and the first dual PFS/OS win for a bispecific ADC in the disease.

• Positive phase III topline results izalontamab brengitecan · Bristol Myers Squibb

Asahi Kasei agreed to buy Germany’s Aicuris for €780m.

The Japanese group is acquiring the antiviral specialist through US subsidiary Veloxis, gaining the Prevymis royalty stream and pritelivir, which met its phase 3 primary endpoint in refractory herpes simplex infection in immunocompromised patients. The deal adds a focused infectious-disease portfolio built around transplant medicine.

• Asahi Kasei to acquire Aicuris to accelerate growth in infectious diseases · Aicuris

Hernexeos won rapid first-line approval in lung cancer, only 44 days after filing.

The FDA granted accelerated approval to Boehringer Ingelheim’s Hernexeos for first line HER2-mutant non-small cell lung cancer, based on a 76% response rate in treatment-naive patients. It is the second approval through the National Priority Voucher route, giving an early indication of how quickly the pathway can move.

• FDA approves Hernexeos for first-line lung cancer · Boehringer Ingelheim

Caris reported interim data on its multi-cancer blood test.

In 2,122 samples from Achieve 1, Caris Detect showed 56.8% sensitivity at stage I, rising to 99.1% at stage IV. Specificity was 99.1% in 121 asymptomatic participants and 95.3% in the 1,505-person undiagnosed population. A blinded validation in about 865 held-out samples is due later in Q1.

• Caris Life Sciences completes interim readout of Achieve 1 study · Caris Life Sciences

Roche unveiled its Axelios sequencer, targeting a $150 human genome.

Roche revealed details of its sequencing-by-expansion chemistry in early 2025. At AGBT it unveiled the instrument, Axelios 1, which is due to launch later this year, targeting a $150 whole human genome at scale. Roche will enter the sequencing market with an instrument, an aggressive cost claim and the balance sheet to compete, adding further pressure on Illumina and its emerging challengers.

Advances in Genome Biology and Technology (AGBT) · Roche

Generate Biomedicines priced its IPO at $400m.

The Flagship-founded protein design company sold 25m shares on Nasdaq at $16. Trading under GENB begins Friday. Its lead asset, GB-0895, is an AI-engineered anti-TSLP antibody already in phase 3 severe asthma trials, with twice-yearly dosing under evaluation. The IPO is therefore a relatively advanced public-market test of generative protein design rather than another preclinical AI platform story.

• Generate Biomedicines announces pricing of IPO · Generate Biomedicines

Friday 27 February

Illumina launched TruPath Genome and detailed its NovaSeq X roadmap at AGBT.

TruPath Genome is designed to compete with long-read sequencing, which Illumina says doubles the throughput of competing long-read methods. The company also outlined an 18-month NovaSeq X roadmap increasing output 40% to 35bn reads per flow cell, alongside Q70 duplex accuracy for specialised high-sensitivity applications.

• Illumina launches TruPath Genome, unveils NovaSeq X roadmap · Illumina

MGI conceded its sequencer infringes Oxford Nanopore’s Australian patents.

MGI accepted in the Federal Court (Australia) that its Cyclone SEQ WT02 infringes four Oxford Nanopore patents, leaving infringement itself undisputed ahead of a 2027 trial on remaining defences and damages. Separate UK High Court proceedings against MGI/BGI entities continue over trade-secret infringement and breach of confidence.

• Update on legal action against MGI Australia · Oxford Nanopore

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• Grail’s Galleri missed its NHS trial primary endpoint, shares fell >45%.

• Danaher and Masimo agreed a deal at about $9.9bn enterprise value.

• An FDA NEJM article suggests one pivotal trial could become the approval default.

• Bayer proposed a $7.25bn Roundup class settlement.

• Compass Pathways’ COMP360 met its second phase 3 primary endpoint.

Browse by day:

• Weekend catch-up: Gilead-Genhouse · Disc Medicine

• Monday: Roche Gazyva · Novartis on the UK

• Tuesday: Danaher-Masimo · Bayer · Compass · Ocular · CSL-Lilly

• Wednesday: FDA one-trial · Moderna

• Thursday: Grail · AZ Calquence · Hims-Eucalyptus · Guardant-MetaSight · Element

Weekend catch-up

Gilead took global rights to a clinic-ready cancer drug GH31 from China’s Genhouse Bio for $80m upfront, in a deal worth up to $1.45bn in milestones.

GH31 is a MAT2A inhibitor, a synthetic lethality approach aimed at MTAP-deleted tumours, with clearance to enter clinical trials in both the US and China. Gilead takes worldwide development and commercialisation. The deal adds to the run of US buyers sourcing clinic-ready oncology assets from Chinese biotechs.

• Global partnership with Gilead on MAT2A inhibitor GH31 · Genhouse Bio

The FDA issued a complete response letter for Disc Medicine’s bitopertin.

The FDA asked for results for the erythropoietic protoporphyria candidate from the continuing APOLLO phase 3 study. The application had carried a Commissioner’s National Priority Voucher, meant to speed favoured drugs through review. The letter resets the timeline on Disc’s lead asset without ending the programme.

• Complete response letter from FDA for bitopertin · Disc Medicine

Monday 16 February

Roche’s Gazyva (obinutuzumab) delivered positive phase 3 results in primary membranous nephropathy.

The results could support the first approval in the kidney disease and further extend use of the oncology antibody into immune-mediated conditions, following its clearance in lupus nephritis last autumn.

• Positive phase III results for Gazyva in primary membranous nephropathy · Roche

Novartis’s UK and Ireland head said the NHS’s reformed approach to evaluating new medicines is “slowly killing the life science sector in the UK”.

Johan Kahlstrom told The Times the changes still do not go far enough. The criticism follows 15 years of Novartis cutting back its UK footprint, which the company attributes to NICE’s cost-effectiveness rules limiting access to its medicines. Novartis has repeatedly described the UK as “uninvestable”.

Novartis’s UK chief says NHS medicines assessment is throttling the sector · The Times

Tuesday 17 February

Danaher agreed to buy Masimo for $9.9bn.

Danaher will pay $180/sh in cash, valuing Masimo at $9.9bn including debt. The deal adds pulse oximetry, sensor technology and AI-enabled patient monitoring to Danaher’s diagnostics portfolio, where Masimo will sit alongside Radiometer, Leica, Cepheid and Beckman Coulter. Danaher expects Masimo to generate >$530m EBITDA in 2027, with >$125m of cost and $50m of revenue synergies by year five. The price is around 18x 2027 EBITDA, falling to 15x including expected synergies. Closing H2 2026.

• Danaher to acquire Masimo Corporation · Danaher

Bayer proposed a $7.25bn settlement of the Roundup litigation.

The Monsanto US class settlement would resolve current and future claims alleging Roundup causes cancer. The litigation has weighed on Bayer since its 2018 acquisition of Monsanto. The proposed settlement would cap a long-running legal exposure that has been difficult to quantify and reduce the risk of years of further litigation.

• Roundup class settlement agreement to resolve current and future claims · Bayer

Compass Pathways’ synthetic psilocybin met its endpoint in a second phase 3 trial.

COMP360, a synthetic formulation of psilocybin, the psychoactive compound found in “magic mushrooms”, met the primary endpoint in the second of two phase 3 trials in treatment-resistant depression. Shares rose about 30%. Compass has requested an FDA meeting to discuss a rolling submission and expects to complete its NDA in Q4.

• Met primary endpoint 2nd phase 3 trial of COMP360 psilocybin · Compass Pathways

Ocular Therapeutix’s AXPAXLI beat a single aflibercept dose in phase 3, but shares fell more than 25%.

SOL-1 compared one dose of the axitinib intravitreal implant with a single 2 mg dose of Eylea in wet AMD after an aflibercept loading phase, rather than with continued on-label anti-VEGF treatment. AXPAXLI met the trial’s superiority endpoint, supporting planned FDA discussions, but the comparator design tempered investor enthusiasm.

• Positive results from SOL-1 phase 3 superiority trial in wet AMD · Ocular Therapeutix

Eli Lilly licensed CSL’s anti-IL-6 antibody clazakizumab for $100m upfront.

Lilly will explore the antibody in kidney disease and other immuno-inflammatory indications, with CSL eligible for additional milestones and royalties. Clazakizumab previously failed a phase 3 trial in organ transplant rejection, and the licence allows CSL to retain some of the economics without fully funding further development alone.

• CSL and Lilly enter licensing agreement for clazakizumab · CSL

The UK government cleared the way for UK Biobank to receive its participants’ GP records.

A data provision notice published on 10 February, picked up this week by The Times, opens coded primary care data in England to consented cohorts such as UK Biobank. For its 500,000 participants, this could double recorded cases of common conditions including arthritis, asthma, dementia and heart failure. The data cover diagnoses, symptoms, medications, referrals and test results, with identifiers removed and consultation notes excluded. Access starts once the NHS agrees data-sharing terms.

• Milestone for health research Government enables access to GP data · UK Biobank

• Decades of medical insight unlocked as UK Biobank gets GP records · The Times

Wednesday 18 February

FDA moves towards one pivotal trial as its default for drug approval.

Commissioner Marty Makary and Vinay Prasad argued in a New England Journal of Medicine perspective that requiring two pivotal trials has become something of a regulatory dogma, and that one adequate well-controlled trial plus confirmatory evidence should instead be the default. FDA already allowed this, but making it the norm would materially change development planning. Formal guidance is still to come.

• One pivotal trial, the new default for FDA approval ending two-trial dogma · NEJM

The FDA reversed itself and will review Moderna’s flu vaccine, a week after refusing to file the application.

After a Type A meeting with CBER, Moderna proposed a revised regulatory pathway for mRNA-1010: full approval in adults aged 50-64 and accelerated approval in ages 65 and over, with an additional post-marketing study in older adults. FDA accepted the amended BLA and set a 5 August PDUFA date, in time for the 2026–27 flu season.

• FDA will initiate review of seasonal influenza vaccine submission · Moderna

Thursday 19 February

Grail’s Galleri missed its primary endpoint in the NHS-Galleri trial.

The study was the largest test so far of whether a single blood test can detect multiple cancers early enough to improve outcomes, and was central to Grail’s UK strategy. It missed its primary endpoint, sending shares down >45% after hours on Thursday.

Grail highlighted a substantial reduction in stage IV diagnoses and a four-fold increase in cancer detection, but the primary endpoint failure weakens the case for NHS adoption and raises fresh questions over the clinical value of multi-cancer early detection (MCED) screening. FDA premarket approval remains under review.

• NHS-Galleri demonstrates substantial reduction in stage IV cancer diagnoses · Grail

The FDA approved AstraZeneca’s Calquence with Venclexta as the first all-oral, fixed-duration combination for first-line chronic lymphocytic leukaemia (CLL).

The clearance covers adults with untreated CLL or small lymphocytic lymphoma (SLL). It gives AZ a fixed-duration front-line regimen in a market where treatment duration has become an important point of differentiation among BTK-based therapies.

• FDA approves acalabrutinib with venetoclax for CLL or SLL · FDA

• Approved as first all-oral, fixed-duration combination in 1st-line CLL · AstraZeneca

Hims & Hers agreed to buy Australian digital health company Eucalyptus for $240m upfront, in a deal worth up to $1.15bn.

The acquisition expands Hims further beyond the US into Australia and Japan and increases its presence in Germany, Canada and UK. Eucalyptus also adds prescribing and fulfilment infrastructure to support Hims’ international weight-loss business.

• To acquire Eucalyptus, accelerating global consumer health vision · Hims & Hers

Guardant disclosed its December acquisition of MCED startup MetaSight.

Terms were $59m upfront, with up to $90m tied to regulatory and commercial milestones. The acquisition adds a multi-cancer early detection programme to a screening portfolio currently centred on blood-based colorectal cancer testing.

• Q4 and full-year 2025 results and 2026 outlook · Guardant Health

Element unveiled VITARI, a new high-throughput sequencing platform.

Element Biosciences has been pushing into territory held by Illumina and Ultima Genomics with its AVITI line, and its new high-throughput instrument takes the fight to the part of the market that drives the majority of sequencing consumables revenue.

• Element introduces VITARI, redefining what sequencing makes possible · Element

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• The FDA refused to review Moderna’s flu shot.

• ICON opened an accounting probe and withdrew its 2025 guidance.

• BridgeBio’s achondroplasia pill met its phase 3 goal.

• Eli Lilly bet up to $2.4bn on in vivo CAR-T.

• Isomorphic Labs new drug engine goes “well beyond AlphaFold 3”.

Browse by day:

• Weekend catch-up: Roche’s MS drug · Lilly-Innovent

• Monday: Lilly-Orna · Novo v Hims · REGENXBIO · DSM-Firmenich · Takeda-Iambic

• Tuesday: Moderna-FDA · AstraZeneca results · CSL · Isomorphic

• Wednesday: Madrigal-Ribo · Keytruda in ovarian cancer

• Thursday: Sanofi CEO · BridgeBio · EU coalition · ICON · US HRT warnings · PTC

• Friday: Novartis Vanrafia

Weekend catch-up

Roche’s oral BTK inhibitor fenebrutinib matched Ocrevus in primary progressive multiple sclerosis (PPMS).

In the 985-patient phase 3 FENtrepid trial, fenebrutinib met its primary endpoint of non-inferiority to Ocrevus (ocrelizumab) and cut risk of disability progression by 12% at 24 weeks. Fatal events ran at 1.4% against 0.2% on Ocrevus. Roche will submit its full phase 3 package once the relapsing-MS study FENhance 1 reads out in H1 2026.

• Fenebrutinib first in over a decade to reduce disability progression in PPMS · Roche

Eli Lilly and Innovent Biologics signed their seventh collaboration, an oncology and immunology alliance worth up to $8.9bn.

Innovent will be paid $350m upfront and is eligible for up to $8.5bn in milestones plus royalties, and will run development to phase 2 proof-of-concept in China while Lilly gets rights everywhere outside Greater China.

• Innovent announces strategic collaboration with Lilly · Innovent Biologics

Monday 9 February

Eli Lilly agreed to buy Orna Therapeutics for up to $2.4bn.

The deal takes Lilly into in vivo CAR-T, where a patient’s own cells are reprogrammed into cell therapies without being removed from the body. Orna’s lead candidate is a CD19-directed in vivo CAR-T for B-cell-driven autoimmune diseases, built on circular RNA. It is one of the largest acquisitions of an in vivo cell-therapy company to date.

• Lilly to acquire Orna Therapeutics to advance cell therapies · Eli Lilly

Novo sued Hims & Hers for patent infringement over compounded semaglutide.

Novo’s complaint accuses Hims of infringing its US patent covering semaglutide, and seeks a permanent injunction and damages. Hims had said over the weekend it would stop offering the compounded pill after the FDA signalled enforcement.

• Novo Nordisk takes legal action against Hims & Hers · Novo Nordisk

The FDA issued a complete response letter for REGENXBIO’s RGX-121, its gene therapy for Hunter syndrome.

REGENXBIO disclosed that the agency had turned down its accelerated-approval filing for the ultra-rare paediatric disease, questioning the trial’s eligibility criteria, its natural-history control and the surrogate endpoint used. The FDA set out requirements, including a new controlled study, that are hard to run in such a small population.

• REGENXBIO announces regulatory update on RGX-121 BLA for MPS II · REGENXBIO

DSM-Firmenich agreed to sell its Animal Nutrition & Health business to CVC at an enterprise value of about €2.2bn including an earn-out of up to €0.5bn.

The Swiss group expects to receive €1.2bn in value after closing, of which roughly €0.6bn is net cash, with it retaining a 20% stake in the unit, which had about €3.5bn of net sales in 2025. The sale, due to close by the end of 2026, largely completes its exit from animal nutrition to concentrate on fragrance, taste and health.

• dsm-firmenich agrees to divest Animal Nutrition & Health to CVC · DSM-Firmenich

Takeda struck a multi-year AI drug discovery deal with Iambic Therapeutics worth >$1.7bn.

Takeda gains access to Iambic’s platform, including its generative model NeuralPLexer, to advance small-molecule programmes in oncology, gastrointestinal disease and inflammation. Iambic takes upfront and technology-access payments and is eligible for milestones that could exceed $1.7bn plus royalties.

• Collaboration with Takeda to advance AI-driven drug design · Iambic Therapeutics

Tuesday 10 February

The FDA refused to review Moderna’s next-generation flu vaccine.

Moderna said the Center for Biologics Evaluation and Research (CBER) had issued a refusal-to-file letter for mRNA-1010, objecting solely to the trial’s comparator arm, a licensed standard-dose flu vaccine, which it said did not reflect the best-available standard of care. Moderna said the letter raised no safety or efficacy concern.

Moderna rejected the reasoning, noting the FDA had reviewed both phase 3 designs before the trials began and that the filing rested on two positive studies in 43,808 participants that met all primary endpoints. It has requested a Type A meeting.

• Refusal-to-file letter from FDA for investigational flu vaccine mRNA-1010 · Moderna

AstraZeneca reported 8% sales growth for 2025 and held its $80bn 2030 target.

Revenues rose 8% at CER (9% AER) to $58.7bn and core earnings per share 11% at CER to $9.16, and chief executive Pascal Soriot reaffirmed the 2030 goal, underpinned by more than 25 planned blockbusters and expansion in the US and China.

• AstraZeneca results: FY and Q4 2025 · AstraZeneca

CSL said CEO Paul McKenzie would retire and, a day later, issued a profit warning.

Company veteran Gordon Naylor, a former Seqirus president, steps in as interim with the plasma and vaccines group under investor pressure. First-half net profit came in at US$401m, down 81% at constant currency, after about US$1.1bn of impairments against CSL Seqirus and CSL Vifor assets tied to falling US vaccine demand.

• Paul McKenzie retires, Gordon Naylor appointed interim CEO · CSL

• CSL half-year results announcement · CSL

Isomorphic Labs unveiled a drug design engine it says goes well beyond AlphaFold 3.

The London-based Alphabet company said its proprietary engine roughly doubles AlphaFold 3’s accuracy on the hardest ligand-binding cases, reaching about 50% on cases with little similarity to training data, against about 23% for AlphaFold 3.

• IL Drug Design Engine unlocks new frontier beyond AlphaFold · Isomorphic Labs

Wednesday 11 February

Madrigal paid $60m upfront for six preclinical siRNA programmes from China’s Ribo, worth up to $4.4bn.

The licence gives Madrigal exclusive global rights to the MASH candidates, built on Ribo’s GalSTAR liver-targeting platform, to combine with its approved MASH drug Rezdiffra (resmetirom). Another deal to build its combination options around Rezdiffra.

• Madrigal expands MASH pipeline with agreement for six siRNA programs · Madrigal

• Ribo & Ribocure announce exclusive global licensing agreement with Madrigal · Ribo

The FDA approved Keytruda in platinum-resistant ovarian cancer, a tumour type the drug had never cracked.

Merck said the agency cleared Keytruda plus paclitaxel with or without Avastin for adults with PD-L1-positive platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer in the second or third line. The FDA cleared Agilent’s PD-L1 IHC 22C3 pharmDx as the companion diagnostic alongside it.

• Keytruda approved in PD-L1-positive platinum-resistant ovarian cancer · Merck

• PD-L1 IHC 22C3 pharmDx approved as companion diagnostic for Keytruda · Agilent

Thursday 12 February

Sanofi named a new chief executive to succeed Paul Hudson.

Sanofi has appointed Belén Garijo, chief executive of Merck KGaA since 2021 and the first woman to lead a DAX40 company, to the top job. Sanofi said Garijo would bring increased rigour to its strategy and R&D, with the growth of Dupixent under scrutiny.

• Belén Garijo to become Chief Executive Officer of Sanofi · Sanofi

BridgeBio’s infigratinib met its phase 3 goal in achondroplasia.

The oral FGFR1-3 selective tyrosine kinase inhibitor improved annualised height velocity by a mean 2.10 cm/year over placebo at week 52 in the PROPEL 3 trial in children (p<0.0001), and met its key secondary on height Z-score. BridgeBio plans US and European filings in H2 2026, setting up a challenge to BioMarin’s Voxzogo.

• Positive phase 3 topline results for oral infigratinib in achondroplasia · BridgeBio

European life sciences investors managing >€24bn launched a coalition.

Novo Holdings, Sofinnova, Forbion, Omega Funds and HealthCap were among the members founding the European Life Sciences Coalition in Brussels, alongside Invest Europe, to press for more capital to scale the region’s biotech companies.

The coalition put Europe at 7% of global life sciences venture capital against 63% for the United States and 14% for China, and noted that 66 of 67 EU biotech companies to list in the past six years chose to do so outside the EU.

• European Life Sciences Coalition launched to strengthen ecosystem · Invest Europe

ICON disclosed an accounting investigation and pulled 2025 guidance.

The CRO said a probe into revenue recognition had preliminarily found revenue in 2023 and 2024 may each have been overstated by less than 2%. It delayed its full-year results to 30 April and said it expects to report one or more material weaknesses.

• Update on timing of results and investigation into accounting practices · ICON

The FDA removed three risk statements from boxed warnings on six menopausal hormone therapy products.

The agency approved labelling changes stripping the warnings on cardiovascular disease, breast cancer and probable dementia from Prometrium, Divigel, Cenestin, Enjuvia, Estring and Bijuva, after a scientific-literature review begun in November 2025.

• FDA approves labelling changes to menopausal hormone therapy products · FDA

PTC Therapeutics withdrew its US filing for the Duchenne drug Translarna.

PTC pulled its NDA resubmission for Translarna in nonsense-mutation DMD after the FDA indicated the data were unlikely to meet its threshold of substantial evidence of effectiveness, This was its third failed attempt to secure US approval for the drug.

• PTC Therapeutics provides regulatory update on Translarna · PTC Therapeutics

Friday 13 February

Novartis said Vanrafia slowed kidney-function decline in IgA nephropathy, though its key secondary endpoint missed significance.

In the phase 3 ALIGN study, Vanrafia showed an eGFR difference over placebo of 2.39 mL/min/1.73 m² at week 136, the final analysis’s key secondary endpoint, that fell short of statistical significance (p=0.057). Vanrafia already holds accelerated US and China approval and Novartis plans to file for approval in 2026 regardless of the miss.

• Phase 3 data support slowing of kidney-function decline in nephropathy · Novartis

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

• Novo and Lilly guided in opposite directions.

• Pfizer’s Metsera injection beat placebo by up to 12.3%.

• Bayer’s asundexian cut strokes 26%.

• The White House launched TrumpRx.

• Eikon Therapeutics and Veradermics both priced upsized IPOs

Browse by day:

• Weekend catch-up: EU CHMP · Lilly’s Pennsylvania plant · US FDA PreCheck

• Monday: Novo · Roche-SanegeneBio · Sanofi · GSK· PacBio-ILMN · PMCPA v Sanofi

• Tuesday: Pfizer-Metsera data · US funding law

• Wednesday: GSK · UK cancer plan · UK-US pharma deal · FTC-Express Scripts

• Thursday: TrumpRx · Novo-Hims · Bayer asundexian · biotech IPOs

Story of the week

A day apart, obesity’s two leaders guided in opposite directions and Novo’s shares fell 27% over the week.

On Tuesday Novo Nordisk forecast adj. 2026 sales and operating profit growth of -5% to -13% CER, citing US pricing pressure and most-favoured-nation deals. The company, which opened up weight-loss, guided to a contraction after growing sales 10% in 2025. The shares fell from $59.4 on Friday to $43.3 by end Thursday.

Then, on Wednesday, Eli Lilly forecast 2026 sales of $80-$83bn, c.25% above 2025, saying tirzepatide (Mounjaro and Zepbound) volumes more than offset the same US pricing pressure Novo had named. Its Q4 revenue rose 43% to $19.3bn.

• Novo Nordisk releases 2026 sales and operating profit outlook · Novo Nordisk

• Lilly reports fourth-quarter 2025 results and provides 2026 guidance · Eli Lilly

Weekend catch-up

Europe’s CHMP backed semaglutide as the first GLP-1 for MASH and opened a data-integrity review of Amgen’s Tavneos.

At its January meeting the Committee for Medicinal Products for Human Use (CHMP) recommended a conditional marketing authorisation for semaglutide (Kayshild) in non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced fibrosis, the first GLP-1 recommended for the indication in the EU.

It also backed Sanofi’s belumosudil (Rezurock) in chronic graft-versus-host disease after re-examination, and referred Amgen’s Tavneos over data integrity questions.

• CHMP meeting highlights, 26-29 January 2026 · European Medicines Agency

Eli Lilly will spend more than $3.5bn on a Pennsylvania plant to make retatrutide and other obesity drugs.

The Lehigh Valley site is the fourth and last of the US factories Lilly promised under its “Lilly in America” programme, and commits capacity to retatrutide, its triple GIP/GLP-1/glucagon agonist, before the drug is approved.

• Lilly selects Pennsylvania for its newest injectable medicine · Eli Lilly

The FDA opened its PreCheck pilot to drugmakers building new US manufacturing plants.

The scheme offers earlier engagement and a more predictable review for companies building domestic facilities, part of the onshoring push behind a 2025 executive order.

• FDA launches PreCheck pilot program · FDA

Monday 2 February

Novo Nordisk’s CagriSema beat semaglutide in a phase 3 type 2 diabetes trial.

In the 2,728-patient phase 3 REIMAGINE 2 trial, CagriSema (cagrilintide and semaglutide) cut HbA1c by 1.91 percentage points and weight by 14.2% at 68 weeks in patients remaining on treatment, against 1.76 points and 10.2% for semaglutide.

• CagriSema superior HbA1c reduction & weight loss in REIMAGINE 2 · Novo Nordisk

Roche paid $200m upfront for global rights to a SanegeneBio RNAi programme.

Genentech takes the licence, worth up to $1.7bn with milestones and tiered royalties, extending Roche’s return to RNA interference after years out of the field. SanegeneBio runs early development before Genentech leads the clinical development.

• SanegeneBio announces RNAi licensing collaboration with Genentech · SanegeneBio

Sanofi’s venglustat passed a phase 3 trial in Gauchers but failed in Fabry disease.

In the LEAP2MONO study, the oral glucosylceramide synthase inhibitor beat enzyme replacement therapy on neurological measures in type 3 Gaucher, whose neurological manifestations have no approved treatment, and Sanofi will file globally. In the PERIDOT study in Fabry, it missed the patient-reported primary endpoint.

Venglustat met all primary endpoints in a phase 3 type 3 Gaucher disease study · Sanofi

GSK handed back rights to Wave’s RNA-editing drug for AATD.

Wave regains WVE-006, one of the first RNA-editing candidates to reach patients, and will fund it alone while it asks the FDA about potential accelerated-approval, with feedback expected in mid-2026. Data from the RestorAATion-2 cohort is due in Q1.

• Wave announces plans to accelerate regulatory path · Wave Life Sciences

Pacific Biosciences sold its short-read sequencing assets to Illumina for $48m.

PacBio built the short-read line through its Omniome buy but paused it last year to concentrate on long reads. The sale, completed on 30 January, hands the market leader another short-read asset and eliminates a potential competitor.

• PacBio completes sale of short-read sequencing assets · Pacific Biosciences

The UK PMCPA ruled Sanofi breached industry code over Pfizer RSV claims.

The Prescription Medicines Code of Practice Authority found Sanofi’s claim that its antibody Beyfortus was more effective than Pfizer’s RSV vaccine Abrysvo was unsubstantiated, and that comments amounted to advertising a prescription medicine.

• Case ruled in breach of Clause 2 · PMCPA

Tuesday 3 February

Pfizer’s obesity injection from its Metsera deal beat placebo by 12.3%.

In the ongoing 64-week VESPER-3 phase 2b maintenance study, the ultra-long-acting GLP-1 MET-097i produced placebo-adjusted weight loss of 10% and 12.3% at week 28 in patients remaining on treatment on the two doses that will go into phase 3. It is the first real read on the $10bn Metsera acquisition.

• Pfizer’s ultra-long-acting injectable GLP-1 RA shows robust weight loss · Pfizer

Trump signed a $1.2tn spending law that ended the federal shutdown.

The Act carries drug provisions the industry has been tracking: a revived rare paediatric disease priority-review voucher programme, reauthorised to 2029, and new Medicare pharmacy-benefit-manager transparency rules. The House passed it 217-214.

• H.R. 7148, the Consolidated Appropriations Act, 2026, signed · The White House

Wednesday 4 February

GSK guided to slower growth in 2026 in Luke Miels’ first outlook as CEO.

Turnover is forecast up 3-5% this year, down on 2025, while GSK held to its target of sales above £40bn by 2031 as its top-selling HIV drugs approach patent expiry. Miels used his first results as CEO to press a more product-focused, launch-led strategy.

• FY 2025 results announcement · GSK

England set out a national cancer plan targeting 75% five-year survival.

The National Cancer Plan for England commits to three in four people diagnosed from 2035 surviving at least five years, which the government puts at about 320,000 additional lives, on the back of earlier diagnosis and faster treatment. It pledges £2.3bn for 9.5 million more diagnostic tests in community centres open 12 hours/day.

• National Cancer Plan for England · Department of Health and Social Care

The UK’s pharma trade deal with the US will cost the Department of Health and Social Care (DHSC) about £1bn over the current Spending Review period.

In a letter to the Commons Science, Innovation and Technology Committee, Lord Vallance said the cost will be funded through allocations made to DHSC at the Spending Review, with front-line services protected. DHSC/NHS/NICE ran the analysis.

• Cost of UK-US pharma deal will be met by DHSC, minister confirms · SIT Committee

The FTC accepted Express Scripts consent agreement to settle insulin-price case.

Under the proposed order, Express Scripts would stop favouring high-list-price drugs where cheaper equivalents exist and would delink its pay from the rebates it negotiates. Changes the FTC estimates would cut patients’ out-of-pocket costs by up to $7bn over ten years. The case accused the largest PBM of inflating insulin list prices.

• Caremark Rx, Zinc Health Services, et al., In the Matter of (Insulin) · FTC

Thursday 5 February

The White House launched TrumpRx, its direct-to-consumer drug site.

President Trump unveiled TrumpRx.gov, listing about 40 medicines at the cash prices manufacturers agreed under his most-favoured-nation deals. The site does not sell drugs but routes patients to each company’s own platform or discount coupons. How much insured patients gain is unclear, since their copays often sit below the cash price.

• Fact sheet: President Trump launches TrumpRx.gov · The White House

Hims & Hers launched a compounded semaglutide pill at $49 for the first month, and Novo Nordisk said it would take legal action.

Hims began selling a compounded semaglutide tablet at $49 for the first month of a five-month plan, with the remaining months at $99, undercutting the branded oral Wegovy. Hims says the product uses a different formulation and delivery system. Novo called it an unapproved “knockoff” and said that it will take legal action.

• H & H expands weight loss portfolio with compounded semaglutide · Hims & Hers

• Novo Nordisk’s statement on the Hims compounded pill · Novo Nordisk

Bayer’s asundexian cut recurrent strokes by 26% in phase 3.

In the >12,300-patient OCEANIC-STROKE trial the oral Factor XIa inhibitor asundexian reduced ischaemic stroke by 26% after a non-cardioembolic stroke or high-risk transient ischaemic attack, with no rise in major bleeding versus placebo. Bayer described it as the first phase 3 of a Factor XIa inhibitor to be completed successfully.

• Asundexian after a non-cardioembolic ischaemic stroke · Bayer

Eikon Therapeutics and Veradermics both priced upsized IPOs.

Eikon, led by Merck & Co veterans and built around super-resolution microscopy, priced 21.18m shares at $18 to list on Nasdaq, for gross proceeds of c.$381m. Hair-loss specialist Veradermics priced a $256.3m NYSE IPO at $17 on 3 February.

• Eikon Therapeutics announces pricing of upsized IPO · Eikon Therapeutics

• Veradermics announces pricing of upsized IPO · Veradermics

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

Top five stories:

• GRAIL filed Galleri for FDA approval.

• AstraZeneca committed $15bn to China.

• Roche reported 22.5% placebo-adjusted weight loss for CT-388.

• Medicare picked 15 more drugs for price talks.

• FDA lifted Intellia’s phase 3 CRISPR trial hold.

Browse by day:

• Weekend: NHS cancer · Guardant injunction · Merck/Revolution talks end

• Monday: UK biotech IPOs · AZ Cambridge · Genmab halt · Elevidys follow-up

• Tuesday: Roche obesity · Waters/BD · Intellia lift · EU/India deal · Medicare · J&J

• Wednesday: Lilly/Seamless · REGENXBIO · Cellares · Danaher · newborns · Yuvezzi

• Thursday: AZ China · Roche · Thermo Fisher · mRNA flu · Galleri · Repertoire/Lilly

• Friday: AstraZeneca/CSPC deal

Weekend catch-up

NHS England launched a national register for people carrying inherited variants that increase cancer risk.

The register covers >100 genes and gives the NHS a route to monitor and recall people for tests.

• Inherited cancer checks programme · NHS England

Patent Court rejects Guardant’s injunction request against Sophia Genetics.

The Paris Local Division refused provisional measures over Sophia’s liquid-biopsy test. It found two patents more likely than not invalid for added matter and said infringement of the third had not been shown with sufficient certainty.

• Final Order, Guardant Health v. Sophia · Unified Patent Court

Merck was no longer in talks to buy Revolution Medicines, The Wall Street Journal reported.

• Merck No Longer in Talks to Buy Revolution · The Wall Street Journal

Monday 26 January

UK drug developers considering IPOs after a four-year domestic biotech drought.

The BioIndustry Association (BIA) told Fierce Biotech that companies were looking at Nasdaq. Baker McKenzie lawyers expected at least two possible London biotech listings in 2026.

• ‘Things are looking up’: British biotechs · Fierce Biotech

AstraZeneca kept its planned £200m Cambridge expansion on hold.

The company said its planning application preserved the option to build rather than restarting the project. The proposed site would employ about 1,000 people.

• AstraZeneca keeps UK research site · Fierce Biotech

Genmab stopped trial enrolment of cancer candidate acquired in $1.8bn ProfoundBio acquisition.

Endpoints reported that the halt affected an early-stage programme. Genmab bought ProfoundBio in 2024 to acquire its antibody-drug conjugate pipeline.

• Genmab halts enrollment · Endpoints

Sarepta said three-year data showed Elevidys slowed the loss of motor function in ambulatory Duchenne patients.

Elevidys (delandistrogene moxeparvovec) produced at least 70% less decline than a propensity-weighted external control on time to rise and the 10-metre walk or run. The follow-up covered 52 patients from EMBARK.

• Sarepta Announces Positive Topline Results · Sarepta

Tuesday 27 January

Roche’s CT-388 produced 22.5% placebo-adjusted weight loss.

The highest once-weekly dose had not reached a weight-loss plateau at 48 weeks. Some 54% of recipients ended below the obesity threshold, against 13% on placebo, and Roche said phase 3 was expected to begin in Q1.

• Roche announces positive Phase II results · Roche

Waters shareholders approved the combination with BD’s Biosciences.

99% of shares represented at the meeting voted in favour. Waters said regulatory approvals were in place and expected the transaction to close on 9 February.

• Shareholders approve combination with BD’s Biosciences · Waters

The FDA lifted the hold on Intellia’s phase 3 MAGNITUDE-2 CRISPR trial.

The decision restarts enrolment of patients with hereditary transthyretin amyloidosis with polyneuropathy. The MAGNITUDE cardiomyopathy trial remained paused.

• Lift of FDA hold on MAGNITUDE-2 Ph3 trial in ATTRv-PN · Intellia

The EU and India concluded negotiations on a free-trade agreement.

Once signed, ratified and in force, the agreement would eliminate or reduce tariffs on >96% of EU goods exports into India, including 11% tariffs on pharmaceuticals.

• EU and India conclude landmark Free Trade Agreement · European Commission

CMS selected 15 medicines for the third Medicare price-negotiation cycle.

The round includes Part B medicines for the first time. Negotiations run through 2026 and the resulting prices take effect on 1 January 2028.

• 15 drugs selected for third Medicare price-negotiation cycle · CMS

The FDA approved J&J’s Darzalex Faspro quadruplet for multiple myeloma.

Darzalex Faspro (daratumumab and hyaluronidase) can now be combined with bortezomib, lenalidomide and dexamethasone in adults ineligible for an autologous stem-cell transplant.

• DARZALEX FASPRO quadruplet approved for transplant-ineligible myeloma · J&J

Wednesday 28 January

Eli Lilly signed a hearing-loss gene-editing deal with Seamless Therapeutics.

Seamless will design programmable recombinases that can make large, precise DNA changes without relying on the cell’s repair machinery. Lilly receives rights to develop the resulting therapies. Potential payments exceed $1.1bn.

• Lilly collaboration · Seamless Therapeutics

The FDA placed two REGENXBIO gene therapies on clinical hold after a brain tumour was found in a treated child.

The five-year-old had received RGX-111 four years earlier. Preliminary analysis found AAV integration associated with PLAG1 overexpression. RGX-121 was also held because the programmes share relevant risks.

• Regulatory update on ultra-rare MPS programmes · REGENXBIO

Cellares raised $257m to expand automated cell-therapy manufacturing.

BlackRock and Eclipse co-led the Series D, taking total capital raised to $612m. Cellares expects clinical manufacturing in H1 2026 and commercial-scale in 2027.

• $257m Series D led by BlackRock and Eclipse · Cellares

Danaher’s Q4 revenue rose 4.5% to $6.8bn.

Full-year revenue increased 3% to $24.6bn. The life-science tools group guided to 3% to 6% core revenue growth for 2026.

• Q4 and full-year 2025 results · Danaher

An NIH-funded study selected seven programmes to assess integration of whole genome sequencing into public health newborn screening.

BEACONS chose Iowa, Minnesota, New York, Oregon, Puerto Rico, South Carolina and Texas. Its gene list targets conditions that can be acted on during a child’s first year.

• Seven sites and final gene list for genomic newborn screening · Ariadne Labs

The FDA approved Tenpoint’s combination eye drop Yuvezzi for presbyopia.

Yuvezzi (carbachol and brimonidine) combines two existing agents in a formulation for adults with blurry close-up vision due to age. Tenpoint plans a Q2 US launch.

• FDA approval of YUVEZZI, 1st combo presbyopia eye drop · Tenpoint Therapeutics

Thursday 29 January

AstraZeneca committed $15bn to China through 2030.

The investment covers research and manufacturing, including cell therapy and radioconjugates. AstraZeneca said it would expand its existing R&D centres in Beijing and Shanghai and increase its Chinese workforce beyond 20,000.

• AstraZeneca invests $15bn in China through 2030 · AstraZeneca

Roche reported 2025 sales growth of 7%

Sales reached CHF 61.5bn +7% CER, or 2% in CHF. Roche forecast mid-single-digit sales growth at constant currencies for 2026.

• 2025 results: sales up 7% · Roche

Thermo Fisher Scientific’s 2025 revenue rose 4% to $44.6bn.

Q4 revenue increased 7% to $12.2bn. Adjusted earnings per share rose 5% for the year. Thermo guided 2026 revenue of $46.0-$47.2bn, representing growth of 4%-6%.

• Q4 and full-year 2025 results · Thermo Fisher

• TMO Q4 2025 earnings call transcript · The Motley Fool

Sanofi deprioritised its next-generation mRNA seasonal flu vaccine programme, Fierce Biotech reported.

Sanofi said it did not anticipate a near-term launch. Its existing protein-based influenza portfolio remains in development and on the market.

• Sanofi scraps attempt to develop mRNA seasonal flu vaccine · Fierce Biotech

GRAIL submitted Galleri for FDA premarket approval.

The final module completes the application for its multi-cancer early-detection (MCED) blood test. The filing draws on PATHFINDER 2 and the NHS-Galleri trial.

• FDA PMA application for the Galleri multi-cancer early detection test · GRAIL

Repertoire and Eli Lilly signed an autoimmune collaboration worth up to $1.9bn plus royalties.

Repertoire receives $85m upfront and up to $1.84bn in milestones. It will use its DECODE platform to nominate tolerising therapies before Lilly takes over clinical development and commercialisation.

• Lilly collaboration on tolerizing therapies for autoimmune diseases · Repertoire

Friday 30 January

AstraZeneca agreed to pay CSPC $1.2bn upfront for eight long-acting obesity and diabetes programmes.

AstraZeneca would receive ex-China rights to the portfolio, led by once-monthly GLP-1/GIP candidate SYH2082. Closing was expected in Q2. CSPC can receive up to $3.5bn in development and regulatory milestones, plus sales payments and royalties.

• AstraZeneca agrees obesity and T2D deal with CSPC · AstraZeneca

ALETH · OPEN ACCESS RESEARCH

aleth.co

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines

• The US Supreme Court agreed to hear key Hikma v. Amarin case.

• GSK agreed to buy RAPT for an estimated $2.2bn.

• AstraZeneca will quit Nasdaq for the NYSE.

• Moderna pulled back from vaccine trials.

• J&J guided 2026 revenue to $100bn.

In this edition

• Weekend catch-up: SCOTUS skinny label; Cordis sale; biotech IPOs

• Monday: Europe pricing push; Valneva withdrawal

• Tuesday: GSK-RAPT deal; ViiV reshuffle; AZ NYSE; melanoma vaccine; Qiagen strategic review

• Wednesday: J&J $100bn; OpenEvidence raise; IntraBio approval

• Thursday: Abbott miss; Corcept ovarian win; Moderna vaccine retreat; Corxel raise; US-WHO exit

• Friday: Sanofi eczema

Weekend catch-up

The US Supreme Court agreed to hear Hikma’s challenge to Amarin over a generic’s “skinny label”.

The court granted certiorari on Friday in Hikma Pharmaceuticals USA Inc. v. Amarin Pharma, Inc. (No. 24-889), taking up the Federal Circuit ruling that let Amarin’s induced-infringement claim proceed even though Hikma had carved the patented cardiovascular indication out of its generic Vascepa (icosapent ethyl) label. This case impacts branded drugs with secondary uses still under patent and could set how far generics can carve a patented use out of their labelling.

• Hikma Pharmaceuticals USA Inc. v. Amarin Pharma, Inc., No. 24-889 - Supreme Court of the United States, docket entry of 16 January 2026

Hellman & Friedman is exploring the sale of med-device maker Cordis at >$9bn, Bloomberg reported.

The PE firm is working with advisers to gauge interest, with about half a dozen listed medtech companies weighing bids, according to the report; talks are early and no sale is assured. H&F bought Cordis, which makes stents and other cardiovascular devices, from Cardinal Health in 2021 for about $1bn.

• H&F Explores $9 Billion-Plus Sale of Medical Device Maker Cordis - Bloomberg

SpyGlass Pharma and Agomab Therapeutics filed Nasdaq IPOs

SpyGlass is an eye-disease company, whose lead asset is a drug-eluting intraocular lens in phase 3 for glaucoma. It filed its S-1 on Friday to trade under the ticker SGP. Immunology developer Agomab Therapeutics filed for its own Nasdaq listing in the same window. Only a handful of drug developers completed sizeable IPOs in 2025, and several 2026 biotech IPOs are taking shape a week after the JPM conference.

• SpyGlass Pharma, Inc. Form S-1

• Agomab Therapeutics NV Form F-1

Monday 19 January

US drugmakers are pressing European governments to accept higher prices

The push follows the MFN pricing deals US companies struck with the Trump administration, which they say force them to raise prices abroad to defend the $700bn US market. Pfizer chief executive Albert Bourla, the first to sign such a deal, told reporters at the JPM conference that faced with cutting the US price to France’s level or pulling out, the company would stop supplying France.

• Financial Times report, 19 January 2026 - Financial Times

Valneva withdrew its chikungunya vaccine after the FDA suspended the shot.

Valneva voluntarily withdrew the US BLA and IND application for Ixchiq, after the FDA suspended the licence in August 2025 following four new reports of serious adverse events; earlier safety concerns had centred mainly on older recipients. The agency has now also placed the IND on clinical hold to investigate a newly reported serious event in a younger adult. This was Valneva’s main commercial launch it its largest market.

• Valneva Provides Update on Chikungunya Vaccine IXCHIQ

• Valneva Announces FDA’s Decision to Suspend License of IXCHIQ In the U.S.

Tuesday 20 January

GSK agreed to buy US biotech RAPT Therapeutics for $2.2bn, its first major deal under new chief executive Luke Miels.

GSK will pay $58 a share in cash, an equity value of $2.2bn and about $1.9bn net of cash, for RAPT’s ozureprubart, a long-acting anti-IgE antibody in phase 2b for the prevention of food-allergy reactions. Its once-quarterly dosing would set it apart from Novartis and Roche’s Xolair (omalizumab), which dominates the anti-IgE class.

GSK enters agreement to acquire RAPT Therapeutics

Pfizer agreed to exit the HIV joint venture ViiV Healthcare as Shionogi committed $2.1bn to raise its holding.

GSK, Pfizer and Shionogi agreed to replace Pfizer’s 11.7% interest in ViiV with new shares issued to Shionogi. Pfizer receives $1.875bn and GSK a $250m special dividend, leaving GSK with 78.3% and Shionogi 21.7%. Pfizer, which warned in December that 2026 revenue will fall, exits a business it helped found while Shionogi doubles down.

• GSK, Pfizer and Shionogi agree on changes to ViiV Healthcare shareholding

AstraZeneca will delist from Nasdaq and move its US listing to the NYSE.

AZ gave notice that it will withdraw its ADSs and debt from Nasdaq, effective after the close on 30 January, with its ordinary shares and debt starting to trade directly on the NYSE from 2 February. This will complete a shareholder-approved plan to harmonise its listing across London, Nasdaq Stockholm and New York.

• AstraZeneca to complete direct listing of shares and US debt securities on NYSE

Moderna and Merck’s personalised cancer vaccine sustained a 49% cut in the risk of melanoma recurrence or death at five years when added to Keytruda.

In the phase 2b KEYNOTE-942 trial, intismeran autogene (mRNA-4157) plus Keytruda reduced recurrence or death by 49% (hazard ratio 0.510) against Keytruda alone in resected high-risk stage III/IV melanoma, matching the benefit seen at three years.

• Moderna & Merck announce 5-yr data for intismeran autogene in melanoma

Qiagen is weighing strategic options, including sale, Bloomberg reported.

The molecular-testing company is working with advisers and has held talks with several possible buyers, including some US strategics, according to the report; its shares rose about 12%. A 2020 Thermo Fisher approach worth roughly $12bn collapsed after an activist opposed it, and the pending exit of chief executive Thierry Bernard is seen as clearing the way.

• Qiagen Is Said to Consider Strategic Options Amid Fresh Interest - Bloomberg

Wednesday 21 January

J&J guided to 2026 revenue of $100bn to $101bn, led by Darzalex.

FY2025 sales rose 6.0% to $94.2bn and Q4 sales 9.1% to $24.6bn. Darzalex, its multiple myeloma antibody, delivered $14.4bn in 2025, with $3.9bn in Q4. J&J said pharma sales topped $60bn for the first time and forecast double-digit growth by 2030.

• Johnson & Johnson reports Q4 and Full-Year 2025 results

Health-AI company OpenEvidence raised $250m, doubling its valuation to $12bn.

The Series D, co-led by Thrive Capital and DST Global, roughly doubled the valuation of three months earlier and takes total funding to c.$700m. OpenEvidence’s clinical-search tool, used widely by US physicians, joins the wave of capital chasing medical AI.

• OpenEvidence Raises $250 Million to Build Medical Superintelligence for Doctors

IntraBio won European approval for Aqneursa in Niemann-Pick type C and a phase 3 win in ataxia-telangiectasia.

The European Commission cleared Aqneursa for the neurological signs of Niemann-Pick type C in patients aged six and over weighing at least 20 kg. Separately, a phase 3 crossover trial in ataxia-telangiectasia, a rare neurodegenerative disease with no approved treatment, showed a statistically significant improvement on the SARA ataxia scale. IntraBio said it would advance regulatory submissions.

• IntraBio Receives EC Approval of AQNEURSA for Niemann-Pick Type C Disease

• IntraBio Announces Positive Pivotal Trial Results of Levacetylleucine for the Treatment of Ataxia-Telangiectasia

The FDA set out draft guidance on using minimal residual disease and complete response to support accelerated approval in multiple myeloma.

The draft recommends MRD negativity in the bone marrow, measured by flow cytometry or next-generation sequencing, and complete response as primary endpoints to support accelerated approval, with clinical benefit confirmed later on survival or progression. Because response rates in myeloma are now so high, traditional endpoints can no longer adequately differentiate between new therapies.

• Minimal Residual Disease and Complete Response in Multiple Myeloma: Use as Endpoints To Support Accelerated Approval - Federal Register

Thursday 22 January

Abbott’s Q4 sales were light and its shares fell sharply.

Q4 sales rose 4.4% to $11.46bn against a consensus of about $11.8bn, as nutrition fell 8.9% and diagnostics slipped 3.6% organically on China’s volume-based procurement. Devices grew 12.3% and pharma 7.0% organically. It guided to 2026 organic sales growth of 6.5-7.5%. Shares fell around 10% on the day.

• Abbott Reports Q4 and Full-Year 2025 Results; Issues 2026 Financial Outlook

Corcept’s relacorilant improved overall survival in a phase 3 ovarian cancer trial.

In the ROSELLA trial, relacorilant plus nab-paclitaxel cut the risk of death by 35% (hazard ratio 0.65, p=0.0004) against nab-paclitaxel alone in platinum-resistant ovarian cancer, with median survival of 16.0 months versus 11.9. The win follows the FDA’s rejection of relacorilant in Cushing’s syndrome; the agency has set a decision on the ovarian filing for 11 July, and Corcept has filed in Europe.

• Overall Survival Primary Endpoint Met in Corcept’s Pivotal Phase 3 ROSELLA Trial of Relacorilant in Patients with Platinum-Resistant Ovarian Cancer

Moderna will not fund new late-stage vaccine trials blaming US hostility.

Speaking to Bloomberg TV at Davos, Bancel said regulatory delays and shrinking official support had made the market too small: “You cannot make a return on investment if you don’t have access to the U.S. market.“ This retreat pulls late-stage funding from the core technology Moderna was built on, as U.S. vaccine policy undergoes a major overhaul under Health Secretary Robert F. Kennedy Jr.

• Moderna Curbs Investment in Vaccine Trials Due to US Backlash - Bloomberg

China-founded Corxel raised $287m to push an oral GLP-1 pill beyond China.

The Series D1, led by RTW Investments and SR One, funds an ongoing US phase 2 in obesity, a planned global phase 2 in type 2 diabetes and phase 3 preparations for CX11, an oral small-molecule GLP-1 receptor agonist Corxel licensed ex-China from Vincentage. It is one of the larger private rounds for an oral incretin as Eli Lilly, Novo Nordisk and several biotechs race into the pill market.

• CORXEL Announces $287 million Series D1 Financing

The United States formally left the World Health Organization.

The exit took effect on Thursday, a year after President Trump ordered it by executive order on his first day back in office and the required 12 months’ notice ran out. Critics warn the departure damages US and global health.

• Fact Sheet: U.S. Withdrawal from the World Health Organization - HHS

• Withdrawing the US From the World Health Organization - The White House

Friday 23 January

Sanofi will file eczema drug amlitelimab despite a mixed set of phase 3 readouts.

The SHORE study met all primary and key secondary endpoints in moderate-to-severe atopic dermatitis, but COAST 2 was mixed: it hit the US co-primary endpoint while missing statistical significance on its EU co-primaries. Sanofi, which has tipped the OX40-ligand antibody as an immunology blockbuster to follow Dupixent (dupilumab), said it will proceed with global submissions in H2 2026.

• Press Release: Sanofi’s amlitelimab confirms its potential in atopic dermatitis

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